DESIGN OF A PHASE 3 STUDY OF AAV-MEDIATED GENE TRANSFER OF ORNITHINE TRANSCARBAMYLASE (OTC) IN PATIENTS WITH LATE-ONSET OTC DEFICIENCY

Autor:	Laura Konczal, Marie Luz Couce, Mireia del Toro, Janneke Langendonk, Andreas Schulze, Joshua Baker, T. Andrew Burrow, Gerald S. Lipshutz, Nicola Longo, Janet Thomas, J. Lawrence Merritt
Rok vydání:	2023
Předmět:	Endocrinology Endocrinology Diabetes and Metabolism Genetics Molecular Biology Biochemistry
Zdroj:	Molecular Genetics and Metabolism. 138:107437
ISSN:	1096-7192
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::348bea1ca29109608d06848166bdb61d https://doi.org/10.1016/j.ymgme.2023.107437 Zobrazit plný text záznamu Full Text from ScienceDirect