Ospedale San Raffaele S.r.l.

Fondazione Telethon

Sangamo Biosciences, Inc.

Deutsches Krebsforschungszentrum

Sangamo Therapeutics, Inc.

Fondazione Telethon Ets

San Raffaele S.r.l.

Angelo Leone Lombardo

Luigi Naldini

Michael C Holmes

Alessio Cantore

Philip D Gregory

Pietro Genovese

Shuyuan Yao

Christof Von Kalle

Manfred Schmidt

Richard Gabriel

Ali Nowrouzi

Anne Arens

Angelo Amabile

Alessandro Migliara

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

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

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

The present invention relates to a method for determining the in vivo localization of double-strand breaks in a host cell, comprising incubating a host cell suspected to comprise DNA double-strand breaks and a linear polynucleotide comprising a known sequence, detecting the in vivo insertion sites of said polynucleotide in the genome of said host cell, and assessing the in vivo localization of double-strand breaks. Further envisaged by the present invention is a method for obtaining an endonuclease with altered in vivo specificity. Finally, the present invention is directed to a kit for determining in vivo specificity of an endonuclease.

Determination of in vivo DNA double-strand break localization and application thereof

Disclosed herein are methods and compositions for targeted integration of one or more copies of a sequence of interest using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain and integrase defective lentiviral donor constructs.

Methods and compositions for targeted integration

Growing community of inventors

Milan, Italy

Angelo Leone Lombardo