Recent Publications

Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates

Paul T. Ranum, Luis Tecedor, Megan S.Keiser, Yong Hong Chen, David E. Leib, Xueyuan Liu, Beverly L. Davidson Mol. Therapy. 2023 Jan 6

DOI: https://doi.org/10.1016/j.ymthe.2022.12.018

Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain

Bryan P. Simpson, Carolyn M Yrigollen, Aleksandar Izda and Beverly Davidson Mol. Therapy. 2023 Jan 6

DOI: https://doi.org/10.1016/j.ymthe.2023.01.004

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*. HGG Advances, September 26, 2022 (*authors contributed equally)

DOI: https://doi.org/10.1016/j.xhgg.2022.100146

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, Beverly L Davidson. PLOS ONE August 29, 2022

DOI: https://doi.org/10.1371/journal.pone.0273710

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. March 2022