Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis
Author:
Funder
Fondazione Telethon Italy
National Tay-Sachs and Allied Diseases
Vaincre les Maladies Lysosomales
Publisher
Elsevier BV
Subject
Neurology
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3. CRISPR/nCas9-Based Genome Editing on GM2 Gangliosidoses Fibroblasts via Non-Viral Vectors;International Journal of Molecular Sciences;2022-09-14
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