Ospedale San Raffaele S.r.l.

Fondazione Telethon

Sangamo Therapeutics, Inc.

Sangamo Biosciences, Inc.

Fondazione Centro San Raffaele Del Monte Tabor

Fondazione Telethon Ets

Deutsches Krebsforschungszentrum

Editas Medicine, Inc.

San Raffaele S.r.l.

Ospedale San Raffaele

Luigi Naldini

Pietro Genovese

Michael C Holmes

Philip D Gregory

Angelo Leone Lombardo

David Paschon

Lei Zhang

Maria Chiara Bonini

Bernhard Rudolf Gentner

Alessio Cantore

Alessandra Biffi

Elena Provasi

Brian Brown

Zulma Magnani

Andrea Annoni

Sara Mastaglio

Maria Grazia Roncarolo

Elisa Vigna

Carrie M Margulies

Shuyuan Yao

Anna Christina Kajaste-rudnitski

Ali Nowrouzi

Michela Milani

Giulia Schiroli

Carolina Petrillo

Alessandro Migliara

Angelo Amabile

Cecillia Cotta-ramusino

Michele De Palma

Erika Zonari

Francesco Boccalatte

Christof Von Kalle

Manfred Schmidt

Richard Gabriel

Anne Arens

Mario Amendola

Mary Anna Lucia Venneri

Giulia Escobar

Adele Mucci

Brian David Brown

An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of CD47 on the surface of the cell.

Virus vector production

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

A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).

Permanent epigenetic gene silencing

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)

An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of MHC-I on the surface of the cell.

Vector production

A gene vector comprising a miRNA sequence target.

Gene vector

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

A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

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

A method of preparing a therapeutic cell population for clinical use from a starting population of cells comprising haematopoietic stem cells, said method comprising separating a population of cells that substantially do not express CD38 but which express CD34 from the starting population of cells, and transducing the separated cell population with a vector to obtain the therapeutic cell population.

Methods for genetic modification of stem cells

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

Growing community of inventors

Milan, Italy

Luigi Naldini