ODP046 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

Here we present the design and early findings from CAH-300 /NCT05101902, a prescreening protocol to identify participants who are potentially eligible for the CAH-301 Phase 1/2 treatment trial with BBP-631, an AAV5-based gene therapy for classic congenital adrenal hyperplasia (CAH) due to 21-hydroxy...

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Detalles Bibliográficos
Autores principales: Bharucha, Kamal, Weinstein, Debra, Kirby, Kathleen, Auchus, Richard, Geffner, Mitchell, Kim, Mimi, Sarafoglou, Kyriakie, Williams, Rachel, Shaywitz, Adam
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Adrenal
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9700285/
http://dx.doi.org/10.1210/jendso/bvac150.125

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