Evolution of Gene Therapy for Inborn Errors of Immunity-Reference-Cited by-同舟云学术

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Evolution of Gene Therapy for Inborn Errors of Immunity

Published:2024-07-01 Issue:7 Volume:178 Page:645
ISSN:2168-6203
Container-title:JAMA Pediatrics
language:en
Short-container-title:JAMA Pediatr

Author:

Somekh Ido¹²,Hendel Ayal³,Somech Raz¹²

Affiliation:

1. Pediatric Department A and the Immunology Service, Jeffrey Modell Foundation Center, Tel-Aviv, Israel

2. Faculty of Medicine, Edmond and Lily Safra Children’s Hospital, Sheba Medical Center, Tel Aviv University, Tel-Aviv, Israel

3. Institute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel

Abstract

This Viewpoint reviews the history and current state of gene therapy for inborn errors of immunity.

Publisher

American Medical Association (AMA)

Link

https://jamanetwork.com/journals/jamapediatrics/articlepdf/2818958/jamapediatrics_somekh_2024_vp_240011_1719260604.18176.pdf

Reference10 articles.

1. Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium.;Thakar;Lancet,2023

2. Gene therapy for inborn errors of immunity: past, present and future.;Fischer;Nat Rev Immunol,2023

3. FDA investigating safety risks in CAR T-cell recipients.;Nelson;Lancet,2023

4. A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders.;Tucci;Nat Commun,2022

5. Gene correction for SCID-X1 in long-term hematopoietic stem cells.;Pavel-Dinu;Nat Commun

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