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Evidence Synthesis of Gene Therapy and Gene Editing from Different Disorders—Implications for Individuals with Rett Syndrome: A Systematic Review

This systematic review and thematic analysis critically evaluated gene therapy trials in amyotrophic lateral sclerosis, haemoglobinopathies, immunodeficiencies, leukodystrophies, lysosomal storage disorders and retinal dystrophies and extrapolated the key clinical findings to individuals with Rett s...

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Detalles Bibliográficos
Autores principales:	Singh, Jatinder, Goodman-Vincent, Ella, Santosh, Paramala
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10219055/ https://www.ncbi.nlm.nih.gov/pubmed/37240368 http://dx.doi.org/10.3390/ijms24109023

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