All Publications
2023
VWA3A-derived ependyma protomer drives increased therapeutic protein secretion into the CSF. Carrell EM, Chen YH, Ranum PT, Coffin SL, Singh LN, Tecedor L, Keiser MG, Hudry E, Hyman BT, Davidson BL Mol Ther Nucleic Acids. 2023 July 14 (Full Text)
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching. Dawicki-McKenna JM, Felix AJ, Waxman EA, Cheng C, Amado DA, Ranum PT, Bogush A, Dungan LV, Maguire JA,, Gagne AL, Heller EA, French DL, Davidson BL, Prosser BL Nat Commun. 2023 May 6 (Full Text)
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates Ranum PT, Tecedor L, Keiser MS, Leib DE, Liu X, Davidson BL Mol. Therapy. 2023 Jan 6 (Full Text)
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain Simpson BP, Yrigollen CM<, Izda A, Davidson BL Mol. Therapy. 2023 Jan 6 (Full Text)
2022
Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing
Fang L, Mas Monteys A, Dürr A, Keiser M, Cheng C, Harapanahalli A, Gonzalez-Alegre P, Davidson BL*, Wang K* (*authors contributed equally) . HGG Advances. 2022 September (Full Text)
Huntington's disease phenotypes are improved via mTORC1 modulation by small molecule therapy Sophie St-Cyr, Daniel D Child, Emilie Giaime, Alicia R Smith, Christine J Pascua, Seung Hahm, Eddine Saiah, Davidson BL. PLOS ONE. 2022 August 29 (Full Text)
Combined overexpression of ATXN1L and mutant ATXN1 knockdown by AAV rescue motor phenotypes and gene signatures in SCA1 mice
Carrell EM, Keiser MS, Robbins AB, Davidson BL. Molecular Therapy: Methods and Clinical Development. 2022 June (Full Text)
DeepRepeat: direct quantification of short tandem repeats on signal data from nanopore sequencing Fang L, Liu Q, Monteys AM, Gonzalez-Alegre P, Davidson BL, Wang K Genome Biology. 2022 April 28. (Full Text)
Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation
Ahrens-Nicklas RC, Tecedor L, Hall AF, Kane O, Chung RJ, Lysenko E, Marsh ED, Stein CS, Davidson BL. Molecular Therapy. 2022 April (Full Text)
An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion
Keller CG, Shin Y, Monteys AM, Renaud N, Beibel M, Teider N, Peters T, Faller T, St-Cyr S, Knehr J, Roma G,Reyes A, Hild M, Lukashev D, Theil D, Dales N, Cha JH, Borowsky B, Dolmetsch R, Davidson BL, Sivasankaran R. Nature Communications. 2022 March (Full Text)
Limited astrocyte-to-neuron conversion in the mouse brain using NeuroD1 overexpression
Leib D, Chen YH, Monteys AM, Davidson BL. Molecular Therapy. 2022 March (Full Text)
Temporal Phenotypic Changes in Huntington’s Disease Models for Preclinical Studies
St-Cyr S, Smith AR, Davidson BL. Journal of Huntington’s Disease. 2022 February (Full Text)
2021
Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain
Keiser MS, Ranum PT, Yrigollen CM, Carrell EM, Smith GR, Muehlmatt AL, Chen YH, Stein JM, Wolf RL, Radaelli E, Lucas II TJ , Gonzalez-Alegre P, Davidson BL. Nature Medicine. 2021 Oct 18. (Abstract)
Design and validation of a multi-point injection technology for MR-guided convection enhanced delivery in the brain. Prezelski K, Keiser M, Stein J, Lucas TH, Davidson B, Gonzalez P, Vitale F. Front Med Technol. 2021 Oct 14. Doi: 10.3389/fmedt.2021.725844 (Full Text)
Regulated control of gene therapies by drug-induced splicing
Monteys AM, Hundley AA, Ranum PT, Tecedor L, Muehlmatt A, Lim E, Lukashev D, Sivasankaran R, Davidson BL. Nature. 2021 Jul 28. Doi:10.1038/s41586-021-03770-2 (Abstract)
Immortalized striatal precursor neurons from Huntington’s disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics.
Akimov SS, Jiang M, Kedaigle AJ, Arbez N, Marque LO, Eddings CR, Ranum PT, Whelan E, Tang A, Wang R, DeVine LR, Talbot CC, Cole RN, Ratovitski T, Davidson BL, Fraenkel E, Ross CA.
Hum Mol Genet. 2021 Jul 22. Doi: 10.1093/hmg/ddab200 (Abstract)
Gene therapy for ALS: A review
Amado DA, Davidson BL.
Molecular Therapy. 2021 April 9. Doi:10.1016/j.ymthe.2021.04.008 (Abstract)
PIAS1 modulates striatal transcription, DNA damage repair, and SUMOylation with relevance to Huntington's disease
Morozko EL, Smith-Geater C, Monteys AM, Pradhan S, Lim RG, Langfelder P, Kachemov M, Hill A, Stocksdale JT, Cullis PR, Wu J, Ochaba J, Miramontes R, Chakraborty A, Hazra TK, Lau A, St-Cyr S, Orellana I, Kopan L, Wang KQ, Yeung S, Leavitt BR, Reidling JC, Yang XW, Steffan JS, Davidson BL, Sarkar PS, Thompson LM.
Proc Natl Acad Sci USA. 2021 Jan 26. Doi: 10.1073/pnas.2021836118 (Full Text)
2020
Regional Variation of Splicing QTLs in Human Brain
Zhang Y, Yang HT, Kadash-Edmondson K, Pan Y, Pan Z, Davidson BL, Xing Y. American Journal of Human Genetics 2020 Aug 6. doi: 10.1016/j.ajhg.2020.06.002 (Full text)
2019
Gene therapy matures to medicines
Davidson BL, Arruda V
Human Molecular Genetics, 2019 Sep 6 doi: 10.1096/hmg/ddz182 (Abstract)
Neuronal network dysfunction precedes storage and neurodegeneration in a lysosomal storage disorder.
Ahrens-Nicklas RC, Tecedor L, Hall AF, Lysenko E, Cohen AS, Davidson BL, Marsh ED.
JCI Insight. 2019 Nov 1. doi: 10.1172/jci.insight.131961 (Abstract)
Doubling down on siRNAs in the brain.
Davidson BL.
Nat Biotechnol. 2019 Aug. doi: 10.1038/s41587-019-0204-1 (Abstract)
Standard screening methods underreport AAV-mediated transduction and gene editing.
Lang JF, Toulmin SA, Brida KL, Eisenlohr LC, Davidson BL Nat Commun, 2019 Jul 30. doi: 10.1038/s41467-019-11321-7. (Full Article https://www.ncbi.nlm.nih.gov/pubmed/31363095) (Full text)
AAV-Mediated Progranulin Delivery to a Mouse Model of Progranulin Deficiency Causes T Cell-Mediated Toxicity
Amado DA, Rieders JM, Diatta F, Hernandez-Con P, Singer A, Mak JT, Zhang J, Lancaster E, Davidson BL, Chen-Plotkin AS
Molecular Therapy 2019 Feb 6. doi: 10.1016/j.ymthe.2018.11.013 (Full text)
CRISPR to the Rescue: Advances in Gene Editing for the FMR1 Gene
Yrigollen CM, Davidson BL
Brain Sciences 2019 Jan 21. doi: 10.3390/brainsci9010017 (Full text)
2018
Adeno-Associated Virus Production, Purification, and Titering.
Chen YH, Keiser MS, Davidson BL
Current Protocols in Mouse Biology 2018 Nov 29. doi: 10.1002/cpmo.56 (Abstract)
Viral Vectors for Gene Transfer.
Chen YH, Keiser MS, Davidson BL
Current Protocols in Mouse Biology 2018 Nov 28. doi: 10.1002/cpmo.58 (Abstract)
The long non-coding RNA NEAT1 is elevated in polyglutamine repeat expansion diseases and protects from disease gene-dependent toxicities.
Cheng C, Spengler RM, Keiser MS, Monteys AM, Rieders JM, Ramachandran S, Davidson BL
Hum Mol Genet 2018 Sep 19. doi: 10.1093/hmg/ddy331 (Abstract)
Cardiac mTORC1 Dysregulation Impacts Stress Adaptation and Survival in Huntington’s Disease
Child D, Lee JH, Pascua JC, Chen YH, Mas Monteys A, Davidson BL
Cell Reports 2018 Apr 24. doi: 10.1016/j.celrep.2018.03.117 (Full text)
Modulating membrane fluidity corrects Batten disease phenotypes in vitro and in vivo
Schultz M, Tecedor L, Lysenko E, Ramachandran S, Stein CS, Davidson BL
Neurobiology of Disease 2018 April 13 doi: 10.1016/j.nbd.2018.04.010 (Full text)
Overcoming limitations inherent in sulfamidase to improve mucopolysaccharidosis IIIA gene therapy
Chen Y, Zheng S, Tecedor L, Davidson BL
Molecular Therapy 2018 Jan 17 doi: 10.1016/j.ymthe.2018.01.010 (Abstract)
2017
CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In vivo
Monteys AM, Ebanks SA, Keiser MS, Davidson BL.
Molecular Therapy 2017 Jan 05. doi: 10.1016/j.ymthe.2016.11.010 (Full text)
2016
RNAi prevents and reverses phenotypes induced by mutant human ataxin-1.
Keiser MS, Monteys AM, Corbau R, Gonzalez-Alegre P, Davidson BL.
Ann Neurol. 2016 Sep 30. doi: 10.1002/ana.24789 (Abstract)
Cis-Acting Single Nucleotide Polymorphisms Alter MicroRNA-Mediated Regulation of Human Brain-Expressed Transcripts.
Ramachandran S, Coffin SL, Tang TY, Jobaliya CD, Spengler RM, Davidson BL.
Hum Mol Genet. 2016 Sep 16. pii: ddw317 (Abstract)
Transcriptome sequencing reveals aberrant alternative splicing in Huntington's disease.
Lin L, Park JW, Ramachandran S, Zhang Y, Tseng YT, Shen S, Waldvogel HJ, Curtis MA, Faull RL, Troncoso JC, Ross CA, Davidson BL, Xing Y.
Hum Mol Genet. 2016 Jul 4. pii: ddw187 (Abstract)
Elucidation of transcriptome-wide microRNA binding sites in human cardiac tissues by Ago2 HITS-CLIP.
Spengler RM, Zhang X, Cheng C, McLendon JM, Skeie JM, Johnson FL, Davidson BL, Boudreau RL
Nucleic Acids Res. 2016 Jul 14. pii: gkw640 (Abstract)
PIAS1 Regulates Mutant Huntingtin Accumulation and Huntington's Disease-Associated Phenotypes In Vivo.
Ochaba J, Monteys AM, O'Rourke JG, Reidling JC, Steffan JS, Davidson BL, Thompson LM.
Neuron. 2016 May 4;90(3):507-20 (Abstract)
2015
AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.
Katz ML, Tecedor L, Chen Y, Williamson BG, Lysenko E, Wininger FA, Young WM, Johnson GC, Whiting RE, Coates JR, Davidson BL.
Sci Transl Med. 2015 Nov 11;7(313):313ra180 (Full text)
Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy.
Keiser MS, Kordower JH, Gonzalez-Alegre P, Davidson BL.
Brain. 2015 Oct 21. pii: awv292. [Epub ahead of print] (Abstract)
Sonic Hedgehog Controls the Phenotypic Fate and Therapeutic Efficacy of Grafted Neural Precursor Cells in a Model of Nigrostriatal Neurodegeneration.
Madhavan L, Daley BF, Davidson BL, Boudreau RL, Lipton JW, Cole-Strauss A, Steece-Collier K, Collier TJ.
PLoS One. 2015 Sep 4 (Abstract)
Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo.
Monteys AM, Wilson MJ, Boudreau RL, Spengler RM, Davidson BL.
Mol Ther Nucleic Acids. 2015 Apr 7 (Abstract)
Reinstating aberrant mTORC1 activity in Huntington's disease mice improves disease phenotypes.
Lee JH, Tecedor L, Chen YH, Monteys AM, Sowada MJ, Thompson LM, Davidson BL.
Neuron. 2015 Jan 21 (Abstract)
2014
Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain.
Monteys AM, Spengler RM, Dufour BD, Wilson MS, Oakley CK, Sowada MJ, McBride JL, Davidson BL.
Nucleic Acids Res. 2014 Dec 1 (Abstract)
Chondroitin Sulfate is the Primary Receptor for a Peptide-Modified AAV That Targets Brain Vascular Endothelium In Vivo.
Geoghegan JC, Keiser NW, Okulist A, Martins I, Wilson MS, Davidson BL.
Mol Ther Nucleic Acids. 2014 Oct 14 (Abstract)
Platelet Activating Factor Receptor Activation Improves siRNA Uptake and RNAi Responses in Well-differentiated Airway Epithelia.
Krishnamurthy S, Behlke MA, Apicella MA, McCray PB Jr, Davidson BL.
Mol Ther Nucleic Acids. 2014 Jul 15 (Abstract)
Retinal transduction profiles by high-capacity viral vectors.
Puppo A, Cesi G, Marrocco E, Piccolo P, Jacca S, Shayakhmetov DM, Parks RJ, Davidson BL, Colloca S, Brunetti-Pierri N, Ng P, Donofrio G, Auricchio A.
Gene Ther. 2014 Jul 3 (Abstract)
Nonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7.
Ramachandran PS, Boudreau RL, Schaefer KA, La Spada AR, Davidson BL.
Mol Ther. 2014 Jun 16. Abstract)
Human-specific microRNA regulation of FOXO1: implications for microRNA recognition element evolution.
McLoughlin HS, Wan J, Spengler RM, Xing Y, Davidson BL.
Hum Mol Genet. 2014 May 15 Abstract)
CLN3 deficient cells display defects in the ARF1-Cdc42 pathway and actin-dependent events.
Schultz ML, Tecedor L, Stein CS, Stamnes MA, Davidson BL.
PLoS One. 2014 May 2 Abstract)
RNA interference-based therapy for spinocerebellar ataxia type 7 retinal degeneration.
Ramachandran PS, Bhattarai S, Singh P, Boudreau RL, Thompson S, Laspada AR, Drack AV, Davidson BL.
PLoS One. 2014 Apr 23 Abstract)
Functional microRNAs and target sites are created by lineage-specific transposition.
Spengler RM, Oakley CK, Davidson BL.
Hum Mol Genet. 2014 Apr 1 Abstract)
Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy.
Keiser MS, Boudreau RL, Davidson BL.
Mol Ther. 2014 Mar;22 Abstract)
Rhes suppression enhances disease phenotypes in Huntington's disease mice.
Lee JH, Sowada MJ, Boudreau RL, Aerts AM, Thedens DR, Nopoulos P, Davidson BL.
J Huntingtons Dis. 2014;3(1) Abstract)
A novel gene delivery method transduces porcine pancreatic duct epithelial cells.
Griffin MA, Restrepo MS, Abu-El-Haija M, Wallen T, Buchanan E, Rokhlina T, Chen YH, McCray PB Jr, Davidson BL, Divekar A, Uc A.
Gene Ther. 2014 Feb;21 Abstract)
Transcriptome-wide discovery of microRNA binding sites in human brain.
Boudreau RL, Jiang P, Gilmore BL, Spengler RM, Tirabassi R, Nelson JA, Ross CA, Xing Y, Davidson BL.
Neuron. 2014 Jan 22 Abstract)
2013
Gene transfer of human Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain.
Hudry E, Dashkoff J, Roe AD, Takeda S, Koffie RM, Hashimoto T, Scheel M, Spires-Jones T, Arbel-Ornath M, Betensky R, Davidson BL, Hyman BT.
Sci Transl Med. 2013 Nov 20 Abstract)
CLN3 loss disturbs membrane microdomain properties and protein transport in brain endothelial cells.
Tecedor L, Stein CS, Schultz ML, Farwanah H, Sandhoff K, Davidson BL.
J Neurosci. 2013 Nov 13 Abstract)
Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice.
Rodríguez-Lebrón E, Costa Mdo C, Luna-Cancalon K, Peron TM, Fischer S, Boudreau RL, Davidson BL, Paulson HL.
Mol Ther. 2013 Oct;21Abstract)
Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease.
Costa Mdo C, Luna-Cancalon K, Fischer S, Ashraf NS, Ouyang M, Dharia RM, Martin-Fishman L, Yang Y, Shakkottai VG, Davidson BL, Rodríguez-Lebrón E, Paulson HL.
Mol Ther. 2013 Oct;21Abstract)
RNAi or overexpression: alternative therapies for Spinocerebellar Ataxia Type 1.
Keiser MS, Geoghegan JC, Boudreau RL, Lennox KA, Davidson BL.
Neurobiol Dis. 2013 AugAbstract)
Recent advances in RNA interference therapeutics for CNS diseases.
Ramachandran PS, Keiser MS, Davidson BL.
Neurotherapeutics. 2013 Jul;10Abstract)
Efficient delivery of RNA interference oligonucleotides to polarized airway epithelia in vitro.
Ramachandran S, Krishnamurthy S, Jacobi AM, Wohlford-Lenane C, Behlke MA, Davidson BL, McCray PB Jr.
Am J Physiol Lung Cell Mol Physiol. 2013 Jul 1 Abstract)
Sustained mobilization of endogenous neural progenitors delays disease progression in a transgenic model of Huntington's disease.
Benraiss A, Toner MJ, Xu Q, Bruel-Jungerman E, Rogers EH, Wang F, Economides AN, Davidson BL, Kageyama R, Nedergaard M, Goldman SA.
Cell Stem Cell. 2013 Jun 6Abstract)
Altered Purkinje cell miRNA expression and SCA1 pathogenesis. Rodriguez-Lebron E, Liu G, Keiser M, Behlke MA, Davidson BL. Neurobiol Dis. 2013 Jan 30. (Abstract)
siSPOTR: a tool for designing highly specific and potent siRNAs for human and mouse. Boudreau RL, Spengler RM, Hylock RH, Kusenda BJ, Davis HA, Eichmann DA, Davidson BL. Nucleic Acids Res. 2013 Jan 7;41(1) (Abstract)
In vivo SELEX for Identification of Brain-penetrating Aptamers. Cheng C, Chen YH, Lennox KA, Behlke MA, Davidson BL. Mol Ther Nucleic Acids. 2013 Jan 8. (Abstract)
2012
Applying gene silencing technology to contraception.
Dissen GA, Lomniczi A, Boudreau RL, Chen YH, Davidson BL, Ojeda SR.
Reprod Domest Anim. 2012 Dec;47 Suppl 6:381-6. (Abstract)
Manipulation of Cell Physiology Enables Gene Silencing in Well-differentiated Airway Epithelia.
Krishnamurthy S, Behlke MA, Ramachandran S, Salem AK, McCray PB Jr, Davidson BL
Mol Ther Nucleic Acids. 2012 Aug 28. (Abstract)
Taking a break from huntingtin.
Davidson BL.
Mol Ther Nucleic Acids. 2012 Aug 21. (Abstract)
Singles engage the RNA interference pathway.
Davidson BL, Monteys AM.
Cell. 2012 Aug 31;150(5):873-5. (Abstract)
Targeted gene silencing to induce permanent sterility.
Dissen GA, Lomniczi A, Boudreau RL, Chen YH, Davidson BL, Ojeda SR.
Reprod Domest Anim. 2012 Aug;47 Suppl 4:228-32. (Abstract)
Dicer is required for proliferation, viability, migration and differentiation in corticoneurogenesis.
McLoughlin HS, Fineberg SK, Ghosh LL, Tecedor L, Davidson BL.
Neuroscience. 2012 Oct 25;223:285-95. (Abstract)
MiR-34a represses Numbl in murine neural progenitor cells and antagonizes neuronal differentiation.
Fineberg SK, Datta P, Stein CS, Davidson BL.
PLoS One. 2012;7(6). (Abstract)
Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease.
Chen YH, Claflin K, Geoghegan JC, Davidson BL.
Mol Ther. 2012 Jul;20(7):1393-9. (Abstract)
Generation of hairpin-based RNAi vectors for biological and therapeutic application.
Boudreau RL, Davidson BL.
Methods Enzymol. 2012;507:275-96. (Abstract)
2011
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease.
McBride JL, Pitzer MR, Boudreau RL, Dufour B, Hobbs T, Ojeda SR, Davidson BL.
Mol Ther. 2011 Dec;19(12):2152-62. (Abstract)
Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease.
Boudreau RL, Spengler RM, Davidson BL.
Mol Ther. 2011 Dec;19(12):2169-77. (Abstract)
Clarifying lysosomal storage diseases.
Schultz ML, Tecedor L, Chang M, Davidson BL.
Trends Neurosci. 2011 Aug;34(8):401-10. (Abstract)
Gene therapy grows into young adulthood: special review issue.
Lee B, Davidson BL.
Hum Mol Genet. 2011 Apr 15;20(R1):R1. (Abstract)
Current prospects for RNA interference-based therapies.
Davidson BL, McCray PB Jr.
Nat Rev Genet. 2011 May;12(5):329-40. (Abstract)
RNAi medicine for the brain: progresses and challenges.
Boudreau RL, Rodríguez-Lebrón E, Davidson BL.
Hum Mol Genet. 2011 Apr 15;20(R1):R21-7. (Abstract)
Widespread establishment and regulatory impact of Alu exons in human genes.
Shen S, Lin L, Cai JJ, Jiang P, Kenkel EJ, Stroik MR, Sato S, Davidson BL, Xing Y.
Proc Natl Acad Sci U S A. 2011 Feb 15;108(7):2837-42. (Abstract)
A knock-in reporter mouse model for Batten disease reveals predominant expression of Cln3 in visual, limbic and subcortical motor structures.
Ding SL, Tecedor L, Stein CS, Davidson BL.
Neurobiol Dis. 2011 Feb;41(2):237-48. (Abstract)
2010
Changes in expression of NMDA-NR1 receptor subunits in the rostral ventromedial medulla modulate pain behaviors.
Da Silva LF, Walder RY, Davidson BL, Wilson SP, Sluka KA.
Pain. 2010 Oct;151(1):155-61. (Abstract)
Evolution of alternative splicing in primate brain transcriptomes.
Lin L, Shen S, Jiang P, Sato S, Davidson BL, Xing Y.
Hum Mol Genet. 2010 Aug 1;19(15):2958-73. (Abstract)
RNAi therapeutics for CNS disorders.
Boudreau RL, Davidson BL.
Brain Res. 2010 Jun 18;1338:112-21. (Abstract)
Osmoregulation of ceroid neuronal lipofuscinosis type 3 in the renal medulla.
Stein CS, Yancey PH, Martins I, Sigmund RD, Stokes JB, Davidson BL.
Am J Physiol Cell Physiol. 2010 Jun;298(6):C1388-400. (Abstract)
Huntington's disease: progress toward effective disease-modifying treatments and a cure.
Johnson CD, Davidson BL.
Hum Mol Genet. 2010 Apr 15;19(R1):R98-R102. (Abstract)
Structure and activity of putative intronic miRNA promoters.
Monteys AM, Spengler RM, Wan J, Tecedor L, Lennox KA, Xing Y, Davidson BL.
RNA. 2010 Mar;16(3):495-505. (Abstract)
2009
Adenosine deamination in human transcripts generates novel microRNA binding sites.
Borchert GM, Gilmore BL, Spengler RM, Xing Y, Lanier W, Bhattacharya D, Davidson BL
Hum Mol Genet. 2009 Dec 15;18(24):4801-7. (Abstract)
MicroRNAs potentiate neural development.
Fineberg SK, Kosik KS, Davidson BL.
Neuron. 2009 Nov 12;64(3):303-9. (Abstract)
Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy.
Chen YH, Chang M, Davidson BL.
Nat Med. 2009 Oct;15(10):1215-8. (Abstract) (pdf)
Comment in:
Gateway to the diseased brain.
Mingozzi F, High KA.
Nat Med. 2009 Oct;15(10):1123-4. (Abstract)
Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice.
Boudreau RL, McBride JL, Martins I, Shen S, Xing Y, Carter BJ, Davidson BL.
Mol Ther. 2009 Jun;17(6):1053-63. (Abstract) (pdf)
Large-scale analysis of exonized mammalian-wide interspersed repeats (MIRs) in primate genomes.
Lin L, Jiang P, Shen S, Sato S, Davidson BL, Xing Y.
Hum Mol Genet. 2009 Jun 15;18(12):2204-14. (Abstract)
Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo.
Boudreau RL, Martins I, Davidson BL.
Mol Ther. 2009 Jan;17(1):169-75. (Abstract) (pdf)
2008
The bifunctional microRNA miR-9/miR-9* regulates REST and CoREST and is downregulated in Huntington's disease.
Packer AN, Xing Y, Harper SQ, Jones L, Davidson BL.
J Neurosci. 2008 Dec 31;28(53):14341-6. (Abstract)
Restoring Acid-sensing ion channel-1a in the amygdala of knock-out mice rescues fear memory but not unconditioned fear responses.
Coryell MW, Wunsch AM, Haenfler JM, Allen JE, McBride JL, Davidson BL, Wemmie JA.
J Neurosci. 2008 Dec 17;28(51):13738-41. (Abstract)
Diverse splicing patterns of exonized Alu elements in human tissues.
Lin L, Shen S, Tye A, Cai JJ, Jiang P, Davidson BL, Xing Y.
PLoS Genet. 2008 Oct 17;4(10):e1000225. (Abstract)
Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs.
Boudreau RL, Monteys AM, Davidson BL.
RNA. 2008 Sep;14(9):1834-44. (Abstract)
Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis.
Chang M, Cooper JD, Sleat DE, Cheng SH, Dodge JC, Passini MA, Lobel P, Davidson BL.
Mol Ther. 2008 Apr;16(4):649-56. (Abstract) (pdf)
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi.
McBride JL, Boudreau RL, Harper SQ, Staber PD, Monteys AM, Martins I, Gilmore BL, Burstein H, Peluso RW, Polisky B, Carter BJ, Davidson BL.
Proc Natl Acad Sci U S A. 2008 Apr 15;105(15):5868-73. (Abstract)
2007
A knock-in reporter model of Batten disease.
Eliason SL, Stein CS, Mao Q, Tecedor L, Ding SL, Gaines DM, Davidson BL.
J Neurosci. 2007 Sep 12;27(37):9826-34. (Abstract)
Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS.
Liu G, Chen YH, He X, Martins I, Heth JA, Chiorini JA, Davidson BL.
Mol Ther. 2007 Feb;15(2):242-7. (Abstract)
RNA interference: a tool for querying nervous system function and an emerging therapy.
Davidson BL, Boudreau RL.
Neuron. 2007 Mar 15;53(6):781-8. (Abstract)
Transvascular delivery of small interfering RNA to the central nervous system.
Kumar P, Wu H, McBride JL, Jung KE, Kim MH, Davidson BL, Lee SK, Shankar P, Manjunath N.
Nature. 2007 Jul 5;448(7149):39-43. (Abstract)
Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease.
Cabrera-Salazar MA, Roskelley EM, Bu J, Hodges BL, Yew N, Dodge JC, Shihabuddin LS, Sohar I, Sleat DE, Scheule RK, Davidson BL, Cheng SH, Lobel P, Passini MA.
Mol Ther. 2007 Oct;15(10):1782-8. (Abstract)
2006
Connecdenn, a novel DENN domain-containing protein of neuronal clathrin-coated vesicles functioning in synaptic vesicle endocytosis.
Allaire PD, Ritter B, Thomas S, Burman JL, Denisov AY, Legendre-Guillemin V, Harper SQ, Davidson BL, Gehring K, McPherson PS.
J Neurosci. 2006 Dec 20;26(51):13202-12. (Abstract)
RNA polymerase III transcribes human microRNAs.
Borchert GM, Lanier W, Davidson BL.
Nat Struct Mol Biol. 2006 Dec;13(12):1097-101. (Abstract)
Optimization of feline immunodeficiency virus vectors for RNA interference.
Harper SQ, Staber PD, Beck CR, Fineberg SK, Stein C, Ochoa D, Davidson BL.
J Virol. 2006 Oct;80(19):9371-80. (Abstract)
Integration site choice of a feline immunodeficiency virus vector.
Kang Y, Moressi CJ, Scheetz TE, Xie L, Tran DT, Casavant TL, Ak P, Benham CJ, Davidson BL, McCray PB Jr.
J Virol. 2006 Sep;80(17):8820-3. (Abstract)
All in the RNA family.
Davidson BL.
Nat Biotechnol. 2006 Aug;24(8):951-2. (Abstract)
A TAT-modified fusion protein efficiently penetrates mouse hypoglossal nuclei from transduced ependyma.
Alisky JM, Xia H, Davidson BL.
Neurosci Lett. 2006 Jun 19;401(1-2):40-3. (Abstract)
Gene therapy for lysosomal storage diseases.
Sands MS, Davidson BL.
Mol Ther. 2006 May;13(5):839-49. (Abstract)
RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases.
Denovan-Wright EM, Davidson BL.
Gene Ther. 2006 Mar;13(6):525-31. (Abstract)
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.
Passini MA, Dodge JC, Bu J, Yang W, Zhao Q, Sondhi D, Hackett NR, Kaminsky SM, Mao Q, Shihabuddin LS, Cheng SH, Sleat DE, Stewart GR, Davidson BL, Lobel P, Crystal RG.
J Neurosci. 2006 Feb 1;26(5):1334-42. (Abstract)
RNAi therapy for neurodegenerative diseases.
Boudreau RL, Davidson BL.
Curr Top Dev Biol. 2006;75:73-92. (Abstract)
2005
Gonzalez-Alegre P, Bode N, Davidson BL, Paulsen HL. Silencing primary dystonia: lentiviral-mediated RNA interference therapy for DYT1 dystonia. J Neurosci 25 (45): 10502-9, 2005. (Abstract)
Davidson BL, Harper SQ. Viral delivery of recombinant short hairpin RNAs. Methods Enzymol 392:145-173, 2005. (Abstract)
Liu G, Martins IH, Wemmie, JA, Chiorini JA, Davidson BL. Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated type 4 vectors. Gene Ther 12 (25):9321-7, 2005. (Abstract)
Stein CS, Martins I, Davidson BL. The lymphocytic choriomeningitis virus envelope glycoprotein targets lentiviral gene transfer vector to neural progenitors in the murine brain. Mol Ther (3):382-9, 2005. (Abstract)
Harper SQ, Staber PD, He X, Eliason SL, Martins IH, Mao Q, Yang L, Kotin RM, Paulson HL, Davidson BL. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. PNAS 102 (16):5820-5825, 2005. (Abstract)
Harper SQ, Davidson BL. Plasmid-based RNA interference: construction of small-hairpin RNA expression vectors. Methods Mol Biol 309:219-236, 2005. (Abstract)
Liu G, Martins IH, Chiorini JA, Davidson BL. Adeno-associated virus type 4 (AAV4) targets ependyma and astrocytes in the subventricular zone and RMS. Gene Ther 12 (20): 1503-8, 2005. (Abstract)
Kang Y, Xie L, Tran DT, Stein CS, Hickey M, Davidson BL, McCray Jr PB. Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer. Blood 1 (106):1552-8 2005. (Abstract)
Miller VM, Nelson RF, Gouvion CM, Williams A, Rodriguez-Lebron E, Harper SQ, Davidson BL, Rebagliati MR, Paulson HL. CHIP suppresses polyglutamine aggregation and toxicity in vitro and in vivo. J Neurosci 25 (40):9152-9161, 2005. (Abstract)
Law LK, Davidson BL. What does it take to bind CAR? Mol Ther 12 (4):599-609, 2005. (Abstract)
Orii KO, Grubb JH, Vogler C, Levy B, Tan Y, Markova K, Davidson BL, Mao Q, Orii T, Kondo N, Sly WS. Defining the pathway for Tat-mediated delivery of beta-glucuronidase in cultured cells and MPS VII mice. Mol Ther 12 (2):345-352, 2005. (Abstract)
Harper SQ, Davidson BL. Plasmid-based RNA interference: construction of small-hairpin RNA expression vectors. Methods Mol Biol 309:219-235, 2005. (Abstract)
2004
Xia H, Mao Q, Eliason SL, Harper SQ, Martins IH, Orr HT, Paulson HL, Yang L, Kotin RM, Davidson BL. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nature Medicine 10(8):775-776, 2004. (Abstract)
Alisky JM, Davidson BL. Towards therapy using RNA interference. Am J Pharmacogenomics 4(1):45-51, 2004. (Abstract)
Davidson BL, Paulson HL. Molecular medicine for the brain: silencing of disease genes with RNA interference. Lancet Neurol 3(3):145-9, 2004. (Abstract)
Alisky JM, Davidson BL. Gene transfer to brain and spinal cord using recombinant adenoviral vectors. Methods Mol Biol 246:91-120, 2004. (Abstract)
Miller VM, Gouvion CM, Davidson BL, Paulson HL. Targeting Alzheimer's disease genes with RNA interference: an efficient strategy for silencing mutant alleles. Nucleic Acids Res 32(2):661-8, 2004. (Abstract)
2003
Davidson BL. Hepatic Diseases- Hitting the Target with Inhibitory RNAs. N Engl J Med: 349:24, 2003. (Abstract)
Lotery A, Yang G, Mullins R, Russell S, Schmidt M, Stone E, Lindbloom J, Chiorini J, Kotin R and Davidson BL. Adeno-Associated virus type 5: Transduction Efficiency and Cell-Type specifity in the Primate Retina. Hum Gene Ther 14:1663-1671, 2003. (Abstract)
Alisky J, Hughes S, and Davidson BL. Transduction of neurons lining the cerebral external capsule in mice with feline immunodeficiency virus based vectors. Neurosci Lett 351:120-124, 2003. (Abstract)
Mao Q, Xia H, and Davidson BL. Intracellular trafficking of CLN3, the protein underlying the childhood neurodegenerative disease, Batten Disease. FEBS Lett 555:351-357, 2003. (Abstract)
Mao Q, Foster BJ, Xia H, and Davidson BL. Membrane topology of CLN3, the protein underlying Batten Disease. FEBS Lett 541:40-46, 2003. (Abstract)
Davidson BL and Breakefield XO. Viral vectors for gene delivery to the nervous system. Nat Rev Neurosci 4(5):353-364, 2003. (Abstract)
Gonzalez-Alegre P, Miller VM, Davidson BL, Paulson HL. Toward therapy for DYT1 dystonia: Allele-specific silencing of mutant Torsin A. Ann Neurol 53(6):781-7, 2003. (Abstract)
Miller VM, Xia H, Marrs GL, Gouvion CM, Lee G, Davidson BL, Paulson HL. Allele-specific silencing of dominant disease genes. PNAS 100(12):7195-7200, 2003. (Abstract)
Sinn PL, Hickey MA, Staber PD, Dylla DE, Jeffers SA, Davidson BL, Sanders DA, McCray PB Jr. Lentivirus vectors pseudotyped with filoviral envelope glycoproteins transduce airway epithelia from the apical surface independently of folate receptor alpha.
J Virol 77(10):5902-5910, 2003. (Abstract)
Kim M, Mao Q, Davidson BL, Wiemer DF. Tripeptide probes for tripeptidyl protease I production via gene transfer. J Med Chem 46(9):1603-8, 2003. (Abstract)
Davidson BL, Chiorini JA. Recombinant adeno-associated viral vector types 4 and 5. Preparation and application for CNS gene transfer. Methods Mol Med. 76:269-285, 2003. (Abstract)
de Perrot M, Fischer S, Liu M, Imai Y, Martins S, Sakiyama S, Tabata T, Bai XH, Waddell TK, Davidson BL, Keshavjee S. Impact of human interleukin-10 on vector-induced inflammation and early graft function in rat lung transplantation. Am J Respir Cell Mol Biol (5):616-25, 2003. (Abstract)
Haskell RE, Hughes SM, Chiorini JA, Alisky JM, Davidson BL. Viral-mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene, TPP I to the mouse central nervous system. Gene Ther 10(1): 34-42, 2003. (Abstract)
2002
Xia H, Mao Q, Paulson HL and Davidson BL. siRNA-mediated gene silencing in vitro and in vivo. Nat Biotech 20: 1006-1010, 2002. (Abstract)
Brooks AI, Stein CS, Hughes SM, Heth J, McCray PB Jr, Sauter SL, Johnston JC, Cory-Slechta DA, Federoff HJ, Davidson BL. Functional correction of established CNS deficits in an animal model of lysosomal storage disease using feline immunodeficiency virus-based vectors. PNAS 99(9):6216-6221, 2002. (Abstract)
Yang GS, Schmidt M, Yan Z, Lindbloom JD, Harding TC, Donahue BA, Engelhardt JF, Kotin RM, Davidson BL. Viral mediated transduction of murine retina with AAV: Effects of viral capsid and genome size. J Virol 76(15):7651-7660, 2002. (Abstract)
Derksen TA, Sauter SL, Davidson BL. Correction of the lysosomal defect in the eye of the ß-glucuronidase deficient mouse using vectors derived from feline immunodeficiency virus. J Gene Med. 4(5): 463-469, 2002. (Abstract)
Kang Y, Stein CS, Heth JA, Sinn PL, Penisten AK, Staber PD, Shen H, Barker CK, Martins I, Sharkey CM, Sanders DA, McCray PBJr, Davidson BL. In vivo gene transfer using a nomprimate lentiviral vector pseudotyped with Ross River virus glycoproteins. J Virol 76(18):9378-9388, 2002. (Abstract)
Stein CS, Davidson BL. Gene transfer to the brain using feline immunodeficiency virus-based lentivirus vectors. Meth Enzymol 346:433-452, 2002. (Abstract)
Alisky JM, van de Wetering CI, Davidson BL. Widespread dispersal of cholera toxin subunit b to brain and spinal cord neurons following systemic delivery. Exp Neurol 178:139-146, 2002. (Abstract)
Lotery AJ, Derksen TA, Russell SR, Mullins RF, Sauter SL, Affatigato LM, Stone EM, Davidson BL. Gene transfer to the non-human primate retina with recombinant feline immunodeficiency virus vectors. Hum Gene Ther 13:689-696, 2002. (Abstract)
Hughes SM, Moussavi-Harami F, Sauter SL, Davidson BL. Viral-mediated gene transfer to mouse primary neural progenitor cells. Mol Ther 5(1):16-24, 2002. (Abstract)
Law L, Davidson BL. Adenovirus serotype 30 fiber does not mediate transduction via the coxsackie adenovirus receptor. J Virol 76(2):656-661, 2002. (Abstract)
Sinnayah P, Lindley TE, Staber PD, Cassell MD, Davidson BL, Davisson RL. Selective gene transfer to key cardiovascular regions of the brain: comparison of two viral vector systems. Hypertension 39(2):603-608, 2002. (Abstract)
Seigel GM, Lotery A, Kummer A, Bernard D, Green NDE, Turmaine M, Derksen T, Nussbaum RL, Davidson BL, Wagner J, Mitchison HM. Retinal pathology and function in a CLN3-knockout mouse model of juvenile ceroid lipofuscinosis (Batten Disease). Mol Cell Neurosci. Mol & Cell Neurosci 19:515-527, 2002. (Abstract)
2001
Xia H, Mao Q, Davidson BL. The HIV Tat protein transduction domain improves the biodistribution of ß-glucuronidase expressed from recombinant viral vectors. Nat Biotech 19:640-644, 2001 (Abstract)
Stein CS, Kang Y, Sauter SL, Townsend K, Staber P, Derksen TA, Martins I, Qian J, Davidson BL, McCray PB Jr. In vivo treatment of hemophilia A and mucopoly-saccharidosis type VII using non-primate lentiviral vectors. Mol Ther 3(6):850-856, 2001 (Abstract)
Chu Y, Heistad DD, Cybulsky MI, Davidson BL. VCAM-1 Expression augments adenovirus-mediated gene transfer. ATVB 21(2):238-242, 2001. (Abstract)
Slepushkin VA, Staber PD, Wang G, McCray PB Jr. Davidson BL. Infection of human airway epithelia with H1N1, H2N2, and H3N2 influenza A virus strains. Mol Ther 3(3):395-402, 2001 (Abstract)
Vasquez EC, Beltz TG, Haskell RE, Johnson RF, Meyrelles SS, Davidson BL, Johnson AK. Adenovirus-mediated gene delivery to cells of the magnocellular hypothalamo-neurohypophyseal system. Exp Neurol 167:260-271, 2001. Abstract)
Rauma T, Kumpumäki S, Anderson R, Davidson BL, Ruotsalainen H, Myllylä R, Hautala T. Adenoviral gene transfer restores lysyl hydroxylase activity in type VII Ehlers-Danlos syndrome. J Invest Dermatol 116(4):602-605, 2001 (Abstract)
2000
Stein CS, Martins I, Davidson BL. Long term reversal of hypercholesterolemia in low density lipoprotein receptor (LDLR)-deficient mice by adenovirus-mediated LDLR gene transfer combined with CD154 blockade. J Gene Med 2:41-51,2000. (Abstract)
Davidson BL, Stein CS, Heth JA, Martins I, Kotin RM, Derksen, TA, Zabner J, Ghodsi A, Chiorini JA. Recombinant adeno-associated virus type 2, 4 and 5 vectors: transduction of variant cell types and regions in the mammalian CNS. Proc Natl Acad Sci, USA 97(7):3428-3432, 2000. (Abstract)
Anderson RD, Haskell RE, Xia H, Roessler BJ, Davidson BL. A simple method for the rapid generation of recombinant adenoviral vectors. Gene Ther 7:1034-1038, 2000. (Abstract)
Alisky JM, Hughes SM, Sauter SL, Jolly DJ, Dubensky TW Jr, Staber PD, Chiorini JA, Davidson BL. Transduction of murine cerebellar neurons with recombinant FIV and AAV5 vectors. NeuroReport 11(12):2669-2673, 2000. (Abstract)
Xia H, Anderson B, Mao Q, Davidson BL. Recombinant human adenovirus: targeting to the human transferrin receptor improves gene transfer to brain microcapillary endothelium. J Virol 74(23):11359-11366, 2000. (Abstract)
Alisky JM, Davidson BL. Gene therapy for amyotrophic lateral sclerosis and other motor neuron diseases. Hum Gene Ther 11(17):2315-2329, 2000. (Abstract)
Haskell RE, Carr CJ, Pearce DA, Bennett MJ, Davidson BL. Batten Disease: Evaluation of CLN3 mutations on protein localization and function. Hum Mol Gen. 9(5):735-744, 2000. (Abstract)
Wang G, Deering C, Macke M, Shao J, Burns R, Blau DM, Holmes KV, Davidson BL, Perlman S, McCray PB Jr. Human coronavirus 229E infects polarized airway epithelia from the apical surface. J Virol 74(19):9234-9239, 2000. (Abstract)
Wang G, Zabner J, Slepushkin V, Deering C, Shao J, Bodner M, Jolly DJ, Davidson BL, McCray PB Jr. Increasing epithelial junction permeability enhances gene transfer to airway epithelia in vivo. Am J Resp Cell & Mol Biol 22(2):129-138, 2000. (Abstract)
Zabner J, Seiler M, Walters R, Kotin RM, Fulgerus W, Davidson BL, Chiorini JA. Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J Virol 74(8):3852-3858, 2000. (Abstract)
Toyoda K, Faraci FM, Russo AF, Davidson BL, Heistad DD. Gene transfer of calcitonin gene-related peptide to cerebral arteries. Am J Physiol Heart Circ Physiol 278(2):H586-594, 2000. (Abstract)
Allamand V, Donahue KM, Straub V, Davisson RL, Davidson BL, Campbell KP. Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice. Gene Ther 7(16):1385-1391, 2000. (Abstract)
Lam EWN, Hammond HM, Zwacka R, Darby CJ, Baumgardner KR, Davidson BL, Oberley TD, Engelhardt JF, Oberley LW. Immunolocalization and adenoviral vector-mediated manganese superoxide dismutase gene transfer to experimental oral tumors. J Dent Res 79:1410-1417, 2000. (Abstract)
Griffith TS, Anderson RD, Davidson BL, Williams RD, Ratliff TL. Adenoviral-mediated transfer of the TNF-related apoptosis-inducing ligand/Apo-2 ligand gene induces tumor cell apoptosis. J Immunol 165:2886-2894, 2000. (Abstract)
Leone P, McPhee SWJ, Janson CG, Davidson BL, Freese A, During MJ. Multi-site partitioned delivery of human tyrosine hydroxylase gene with phenotypic recovery in Parkinsonian rats. NeuroReport 11:1145-1151, 2000. (Abstract)
Durbeej M, Cohn RD, Hrstka RF, Moore SA, Allamand V, Davidson BL, Williamson RA, Campbell KP. Disruption of the ß-sarcoglycan gene reveals pathogenetic complexity of limb-girdle muscular dystrophy type 2E. Mol Cell 5(1):141-151, 2000. (Abstract)