A Brazilian patient with late infantile metachromatic leukodystrophy treated with lentiviral hematopoietic stem-cell gene therapy: A report from prenatal diagnosis to early treatment

Autor:	Larissa Faqueti, Gabrielle Iop, Layzon Antonio Lemos da Silva, Francyne Kubaski, Henrique B.L. Borges, Alice Brinckmann Oliveira Netto, Ana C. Brusius-Facchin, Sandra Leistner-Segal, Rejane Gus, Maria Teresa V. Sanseverino, Kristiane Michelin-Tirelli, Fernanda Bender Pasetto, Fernanda Medeiros Sebastiao, Thiago Oliveira Silva, José Antonio Azevedo Magalhães, Carolina Fischinger Moura de Souza, Salvatore Recupero, Valeria Calbi, Francesca Fumagalli, Alessandro Aiuti, Roberto Giugliani
Rok vydání:	2023
Předmět:	Endocrinology Endocrinology Diabetes and Metabolism Genetics Molecular Biology Biochemistry
Zdroj:	Molecular Genetics and Metabolism. 138:107094
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2022.107094
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::9ea8efcfa626873bcb1a6a971d1ef529 https://doi.org/10.1016/j.ymgme.2022.107094 Zobrazit plný text záznamu Full Text from ScienceDirect