Clinical Applications of Gene Therapy for Primary Immunodeficiencies-Reference-Cited by-同舟云学术

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Clinical Applications of Gene Therapy for Primary Immunodeficiencies

Published:2015-04 Issue:4 Volume:26 Page:210-219
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Cicalese Maria Pia¹²,Aiuti Alessandro¹²³

Affiliation:

1. San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, 20132 Milan, Italy.

2. Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, 20132 Milan, Italy.

3. Vita-Salute San Raffaele University, 20132 Milan, Italy.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2015.047

Reference121 articles.

1. Laboratory Diagnosis of Primary Immunodeficiencies

2. Current progress on gene therapy for primary immunodeficiencies

3. Gene therapy for PIDs: Progress, pitfalls and prospects

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2. CRISPR-Cas9 engineering of the RAG2 locus via complete coding sequence replacement for therapeutic applications;Nature Communications;2023-10-27

3. Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs;Molecular Therapy - Nucleic Acids;2023-03

4. CRISPR-Cas9 RAG2 Correction via Coding Sequence Replacement to Preserve Endogenous Gene Regulation and Locus Structure;2023-02-17

5. Multiplex HDR for Disease and Correction Modeling of SCID by CRISPR Genome Editing in Human HSPCs;2022-10-07

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