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Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders
A new approach is adopted to treat primary immunodeficiency disorders, such as the severe combined immunodeficiency (SCID; e.g., adenosine deaminase SCID [ADA-SCID] and IL-2 receptor X-linked severe combined immunodeficiency [SCID-X1]). The success, along with the feasibility of gene therapy, is und...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
De Gruyter
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8093481/ https://www.ncbi.nlm.nih.gov/pubmed/33987480 http://dx.doi.org/10.1515/biol-2021-0033 |