Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My Custom Filters

Results by year

Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2022 6
2023 9
2024 19
2025 22
2026 8

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

53 results

Results by year

Filters applied: . Clear all
Page 1
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
Pipe SW, Leebeek FWG, Recht M, Key NS, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O'Connell N, Pasi KJ, Hart DP, Kazmi R, Astermark J, Hermans CRJR, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary SE, Escobar M, Gomez E, Kruse-Jarres R, Quon DV, Symington E, Wang M, Wheeler AP, Gut R, Liu YP, Dolmetsch RE, Cooper DL, Li Y, Goldstein B, Monahan PE. Pipe SW, et al. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.
Reiss UM, Davidoff AM, Tuddenham EGD, Chowdary P, McIntosh J, Muczynski V, Journou M, Simini G, Ireland L, Mohamed S, Riddell A, Pie AJ, Hall A, Quaglia A, Mangles S, Mahlangu J, Haley K, Recht M, Shen YM, Halka KG, Fortner G, Morton CL, Gu Z, Hayden RT, Neufeld EJ, Okhomina VI, Kang G, Nathwani AC. Reiss UM, et al. N Engl J Med. 2025 Jun 12;392(22):2226-2234. doi: 10.1056/NEJMoa2414783. N Engl J Med. 2025. PMID: 40499172 Free PMC article. Clinical Trial.
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.
Cuker A, Kavakli K, Frenzel L, Wang JD, Astermark J, Cerqueira MH, Iorio A, Katsarou-Fasouli O, Klamroth R, Shapiro AD, Hermans C, Ishiguro A, Leavitt AD, Oldenburg JB, Ozelo MC, Teitel J, Biondo F, Fang A, Fuiman J, McKay J, Sun P, Rasko JEJ, Rupon J; BENEGENE-2 Trial Investigators. Cuker A, et al. N Engl J Med. 2024 Sep 26;391(12):1108-1118. doi: 10.1056/NEJMoa2302982. N Engl J Med. 2024. PMID: 39321362 Clinical Trial.
Fidanacogene Elaparvovec for Hemophilia B - A Multiyear Follow-up Study.
Rasko JEJ, Samelson-Jones BJ, George LA, Giermasz A, Ducore JM, Teitel JM, McGuinn CE, High KA, de Jong YP, Chhabra A, O'Brien A, Smith LM, Winburn I, Rupon J. Rasko JEJ, et al. N Engl J Med. 2025 Apr 17;392(15):1508-1517. doi: 10.1056/NEJMoa2307159. N Engl J Med. 2025. PMID: 40239068 Clinical Trial.
Structural, functional, and immunogenicity implications of F9 gene recoding.
Katneni UK, Alexaki A, Hunt RC, Hamasaki-Katagiri N, Hettiarachchi GK, Kames JM, McGill JR, Holcomb DD, Athey JC, Lin B, Parunov LA, Kafri T, Lu Q, Peters R, Ovanesov MV, Freedberg DI, Bar H, Komar AA, Sauna ZE, Kimchi-Sarfaty C. Katneni UK, et al. Blood Adv. 2022 Jul 12;6(13):3932-3944. doi: 10.1182/bloodadvances.2022007094. Blood Adv. 2022. PMID: 35413099 Free PMC article.
Gene-ius at work: Hemophilia B treatment enters a new era.
Northington MW, Rice SE, Holmes AL, Watts Alexander CS. Northington MW, et al. Am J Health Syst Pharm. 2025 Sep 9;82(18):960-969. doi: 10.1093/ajhp/zxaf005. Am J Health Syst Pharm. 2025. PMID: 39868419 Review.
53 results