Ospedale San Raffaele S.r.l.

Sangamo Therapeutics, Inc.

Sangamo Biosciences, Inc.

Fondazione Telethon

Editas Medicine, Inc.

Fondazione Telethon Ets

Ospedale San Raffaele

Pietro Genovese

Luigi Naldini

Michael C Holmes

Philip D Gregory

David Paschon

Lei Zhang

Maria Chiara Bonini

Elena Provasi

Zulma Magnani

Sara Mastaglio

Angelo Leone Lombardo

Bernhard Rudolf Gentner

Carrie M Margulies

Anna Christina Kajaste-rudnitski

Cecillia Cotta-ramusino

Giulia Schiroli

Carolina Petrillo

Disclosed herein are genome editing systems and related methods which allow for treatment of Hyper IgM Syndrome, a group of disorders characterized by defective CD40 signaling. The compositions and methods described herein rely on the use of donor templates comprising a CD40L exons to restore proper CD40 signaling and B cell class switch recombination.

Systems and methods for treating hyper-igm syndrome

Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.

Method for improving retroviral transduction and gene editing in hematopoietic stem cells using cyclosporine h (CsH)

Disclosed herein are methods and compositions for inactivating TCR genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.

Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases

Disclosed herein are methods and compositions for modifying TCR genes, using nucleases (zinc finger nucleases or TAL nucleases) to modify TCR genes.

The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.

Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells

Targeted disruption of T cell receptor genes using talens

Growing community of inventors

Milan, Italy

Pietro Genovese