التفاصيل البيبلوغرافية
العنوان:
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
المؤلفون:
John M. Cunningham , Yunyu Spence , Arnulfo Jaquilmac Pie , Susan Sleep , Savita Rangarajan , Andrew M. Davidoff , Keith Smith , Cecilia Rosales , Chris Harrington , David C. Linch , Catherine Y.C. Ng , Edward G. D. Tuddenham , Pratima Chowdary , Christopher L. Morton , Mark A. Kay , Etiena Basner-Tschakarjan , Jenny McIntosh , Bertil Glader , Amit C. Nathwani , John T. Gray , Junfang Zhou , Ulrike M. Reiss , John Pasi , James A. Allay , Pradip Rustagi , Federico Mingozzi , James O'Beirne , Arthur W. Nienhuis , John Coleman , Anne Riddell , Deokumar Srivastava , Katherine A. High
سنة النشر:
2011
مصطلحات موضوعية:
medicine.medical_specialty , business.industry , Transgene , Genetic enhancement , Genetic Vectors , General Medicine , Genetic Therapy , Dependovirus , Asymptomatic , Gastroenterology , Hemophilia B , Virus , Article , Viral vector , Alipogene tiparvovec , Factor IX , Internal medicine , Immunology , medicine , Humans , Vector (molecular biology) , medicine.symptom , business , medicine.drug
الوصف:
Background Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder. Methods We infused a single dose of a serotype-8–pseudotyped, self-complementary adeno virus-associated virus (AAV) vector expressing a codon-optimized human factor IX (FIX) transgene (scAAV2/8-LP1-hFIXco) in a peripheral vein in six patients with severe hemophilia B (FIX activity
اللغة:
English
الوصول الحر:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::39e983fb3d15006128dc0ba1780e55a7Test https://europepmc.org/articles/PMC3265081Test /
حقوق:
OPEN
رقم الانضمام:
edsair.doi.dedup.....39e983fb3d15006128dc0ba1780e55a7
قاعدة البيانات:
OpenAIRE
