AAV gene therapy for GBA1-related diseases
Year: 2025
Type: article
Language: en
Topic: Lysosomal Storage Disorders Research
Authors and affiliations:
raw_author_name: Swathi Ayloo
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451
raw_author_name: Jae Cheon Ryu
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451
raw_author_name: Shih-Ching Chou
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451
raw_author_name: Lydia Blatnik
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451
raw_author_name: Erik Wischhof
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451
raw_author_name: Jie Bu
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451
raw_author_name: Lilu Guo
raw_affiliation_strings: Translational Medicine Unit, Sanofi, Cambridge, MA 02141, USA
Translational Medicine Unit, Sanofi, Cambridge, MA, USA, 02141
raw_author_name: Mahmud Hossain
raw_affiliation_strings: Translational Medicine Unit, Sanofi, Cambridge, MA 02141, USA
Translational Medicine Unit, Sanofi, Cambridge, MA, USA, 02141
raw_author_name: Dhiman Ghosh
raw_affiliation_strings: Translational Medicine Unit, Sanofi, Cambridge, MA 02141, USA
Translational Medicine Unit, Sanofi, Cambridge, MA, USA, 02141
raw_author_name: William McCarty
raw_affiliation_strings: Quantitative Pharmacology, Sanofi, Cambridge, MA 02141, USA
Quantitative Pharmacology, Sanofi, Cambridge, MA, USA, 02141
raw_author_name: Ann Byrne
raw_affiliation_strings: Target Disease and Systems Biology, Cambridge, MA 02141, USA
Target Disease and Systems Biology, Cambridge, MA, USA, 02141
raw_author_name: Lilly Chai
raw_affiliation_strings: Target Disease and Systems Biology, Cambridge, MA 02141, USA
Target Disease and Systems Biology, Cambridge, MA, USA, 02141
raw_author_name: Jennifer Clarke
raw_affiliation_strings: Rare Diseases TA, Sanofi, Cambridge, MA 02141, USA
Rare Diseases TA, Sanofi, Cambridge, MA, USA, 02141
raw_author_name: Can Kayatekin
raw_affiliation_strings: Rare Diseases TA, Sanofi, Cambridge, MA 02141, USA
Rare Diseases TA, Sanofi, Cambridge, MA, USA, 02141
raw_author_name: Bailin Zhang
raw_affiliation_strings: Translational Medicine Unit, Sanofi, Cambridge, MA 02141, USA
Translational Medicine Unit, Sanofi, Cambridge, MA, USA, 02141
raw_author_name: S. Pablo Sardi
raw_affiliation_strings: Rare Diseases TA, Sanofi, Cambridge, MA 02141, USA
Rare Diseases TA, Sanofi, Cambridge, MA, USA, 02141
raw_author_name: Martin Goulet
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451
raw_author_name: Bradford Elmer
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451
raw_author_name: Christian Mueller
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA. Electronic address: gmu@sanofi.com
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451. Electronic address: gmu@sanofi.com
raw_author_name: Shyam Ramachandran
raw_affiliation_strings: Genomic Medicine Unit, Sanofi, Waltham, MA 02451, USA
Genomic Medicine Unit, Sanofi, Waltham, MA, USA, 02451