The GuardOne clinical trial: A first-in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease

Autor:	Josie Yang, Aneal Khan, Christopher E. Mason, Richard Pfeifer, Rachel Ciotti, Leslie Jacobsen, Julie A. Kerner, Azadeh Golipour
Rok vydání:	2021
Předmět:	Oncology medicine.medical_specialty business.industry Endocrinology Diabetes and Metabolism Genetic enhancement Disease First in human Biochemistry Viral vector Clinical trial Endocrinology Internal medicine Genetics medicine Open label business Molecular Biology Ex vivo
Zdroj:	Molecular Genetics and Metabolism. 132:S51-S52
ISSN:	1096-7192
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::96202ab84ab88ff31e5ee4d7001d7653 https://doi.org/10.1016/j.ymgme.2020.12.112 Zobrazit plný text záznamu Full Text from ScienceDirect