Open AccessODP046 Initial Lessons from a Prescreening Protocol to Identify Participants with Classic CAH Potentially Eligible for Gene Therapy Treatment with BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
Author(s) -
Kamal Bharucha,
Debra F. Weinstein,
Kathleen Kirby,
Richard J. Auchus,
Mitchell E. Geffner,
Mimi S. Kim,
Kyriakie Sarafoglou,
Rachel Williams,
Adam J. Shaywitz
Publication year - 2022
Publication title -
journal of the endocrine society
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.046
H-Index - 20
ISSN - 2472-1972
DOI - 10.1210/jendso/bvac150.125
Subject(s) - congenital adrenal hyperplasia , glucocorticoid , medicine , mineralocorticoid , genetic enhancement , pediatrics , endocrinology , biology , gene , genetics
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