Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 73 publications
Fidanacogene Elaparvovec for Hemophilia B - A Multiyear Follow-up Study.
Journal: The New England journal of medicine
Published: April 16, 2025
Gene therapy for hemophilia - From basic science to first approvals of "one-and-done" therapies.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: March 06, 2025
Validation of a quantitative cell-based relative potency assay for LUXTURNA.
Journal: Molecular therapy. Methods & clinical development
Published: September 30, 2024
Roadmap for direct and indirect translation of optogenetics into discoveries and therapies for humans.
Journal: Nature neuroscience
Published: January 26, 2024
Gene and cell therapy in 2023: Rich pipeline, slimming resources?
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: December 05, 2023
Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec.
Journal: Blood advances
Published: November 16, 2023
THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE.
Journal: Transactions of the American Clinical and Climatological Association
Published: September 13, 2023
Trial by "Firsts": Clinical Trial Design and Regulatory Considerations in the Development and Approval of the First AAV Gene Therapy Product in the United States.
Journal: Cold Spring Harbor perspectives in medicine
Published: September 12, 2022
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
Journal: The New England journal of medicine
Published: November 17, 2021
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A.
Journal: Molecular therapy. Methods & clinical development
Published: March 11, 2021
Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years.
Journal: Ophthalmology
Published: November 18, 2020
Last Updated: 09/26/2026