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Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Chowdary P, et al. Among authors: long a. N Engl J Med. 2022 Jul 21;387(3):237-247. doi: 10.1056/NEJMoa2119913. N Engl J Med. 2022. PMID: 35857660 Free article. Clinical Trial.
BACKGROUND: FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B. ...After 26 weeks, patients …
BACKGROUND: FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a syn …
A famsin-glucagon axis mediates glucose homeostasis.
Long A, Wang Y, Guo Y, Hong J, Ning G, Meng Z, Wang J, Wang Y. Long A, et al. Cell Metab. 2024 Dec 12:S1550-4131(24)00454-6. doi: 10.1016/j.cmet.2024.11.008. Online ahead of print. Cell Metab. 2024. PMID: 39706194
1,924 results