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Summary M2 gene therapy 2019/2020

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Introduction AAV Oncolytic virus RNA interference Retrovirus Lentivirus Adenovirus Non viral carriers Electrotransfer CRISPR SCID X1 Gene therapy Hematopoietic cells collection Endothelial cells and vascular reconstitution Bioengineered cardiac tissue Exon splicing in neuro-muscular disease Gene therapy for hemoglobin disorders Gene therapy for neuromuscular disorders Viral gene transfer to CNS Ocular gene therapy Ex vivo generation of RBC for transfusion purposes Targeted therapies in rheumatic inflammatory disorders Gene therapy for pancreatic cancer PSC for skin and corneal pathologies SC potential for retinal diseases

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