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New Indications for Hematopoietic Stem Cell Gene Therapy in Lysosomal Storage Disorders

Lysosomal storage disorders (LSDs) are a heterogenous group of disorders due to genetically determined deficits of lysosomal enzymes. The specific molecular mechanism and disease phenotype depends on the type of storage material. Several disorders affect the brain resulting in severe clinical manife...

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Detalles Bibliográficos
Autores principales:	Rossini, Linda, Durante, Caterina, Marzollo, Antonio, Biffi, Alessandra
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2022
Materias:	Oncology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9136164/ https://www.ncbi.nlm.nih.gov/pubmed/35646708 http://dx.doi.org/10.3389/fonc.2022.885639

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