Molecular therapy : the journal of the American Society of Gene Therapy

Gene-corrected immune cell therapy for IL2RA deficiency

Updated

Abstract

Essence

CRISPR-corrected autologous Tregs may restore IL2RA expression and suppressive function for IL2RA deficiency.

Evidence

This ex vivo cell-therapy manufacturing study corrected patient-derived Tregs with CRISPR-Cas9 homology-directed repair and tested IL2RA expression and in vitro suppressive function.

Caveat

The work demonstrates GMP-compatible manufacturing and cell function, not clinical safety, engraftment, or autoimmune disease control after infusion.

Simplified

Full Text

Full text is available at the source.

Funding

Competing interests

Declaration of interests A.M. is a cofounder of Site Tx, Arsenal Biosciences, and Survey Genomics; serves on the boards of directors at Site Tx and Survey Genomics; is a member of the scientific advisory boards of Site Tx, Arsenal Biosciences, Cellanome, Survey Genomics, NewLimit, Network.bio, Amgen, and Tenaya; owns stock in Arsenal Biosciences, Site Tx, Cellanome, NewLimit, Survey Genomics, Network.bio, Tenaya and Lightcast; and has received fees from Site Tx, Arsenal Biosciences, Cellanome, Spotlight Therapeutics, NewLimit, Network.bio, Amgen, Abbvie, Gilead, Pfizer, 23andMe, PACT Pharma, Juno Therapeutics, Tenaya, Lightcast, Trizell, Vertex, Merck, Genentech, GLG, ClearView Healthcare, AlphaSights, Rupert Case Management, Bernstein, and ALDA. He is an investor in and informal advisor to Offline Ventures and a client of EPIQ. The Marson laboratory has received research support from the Parker Institute for Cancer Immunotherapy, the Emerson Collective, Arc Institute, Juno Therapeutics, Epinomics, Sanofi, GlaxoSmithKline, Gilead, and Anthem and reagents from Genscript, 10X, Ultima, Illumina, and Cellanome. A.H. is the founder and chief scientific officer of Cassidy Bio. Q.T. is a co-founder, shareholder, and scientific advisor of Sonoma Biotherapeutics and is a consultant of eGenesis and Qihan Bio. All declared interests had no input in the design, execution, interpretation, or publication of the work in this paper.
PubMed

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