TY  - GEN
AU  - Byrne, Barry J
AU  - Flanigan, Kevin M
AU  - Matesanz, Susan E
AU  - Finkel, Richard S
AU  - Waldrop, Megan A
AU  - D'Ambrosio, Eleonora S
AU  - Johnson, Nicholas E
AU  - Smith, Barbara K
AU  - Bönnemann, Carsten
AU  - Carrig, Sean
AU  - Rossano, Joseph W
AU  - Greenberg, Barry
AU  - Lalaguna, Laura
AU  - Lara-Pezzi, Enrique
AU  - Subramony, Sub
AU  - Corti, Manuela
AU  - Mercado-Rodriguez, Claudia
AU  - Leon-Astudillo, Carmen
AU  - Ahrens-Nicklas, Rebecca
AU  - Bharucha-Goebel, Diana
AU  - Gao, Guangping
AU  - Gessler, Dominic J
AU  - Hwu, Wuh-Liang
AU  - Chien, Yin-Hsiu
AU  - Lee, Ni-Chung
AU  - Boye, Sanford L
AU  - Boye, Shannon E
AU  - George, Lindsey A
PY  - 2025
UR  - https://hdl.handle.net/20.500.12105/27308
AB  - Currently, there are an estimated 8,000 genetic disorders that cumulatively affect approximately 10% of the population. Even among the 5% of patients with genetic disease that have treatment options, these therapeutics rarely address the underlying...
LA  - eng
PB  - Cell Press
KW  - adeno-associated viral vectors
KW  - in vivo gene therapy
TI  - Current clinical applications of AAV-mediated gene therapy.
TY  - research article
ER  -