University of California

National Institute of Transplantation Foundation

University of Southern California

Sangamo Biosciences, Inc.

Other

Denovo Biopharma LLC

Sangamo Therapeutics, Inc.

Tocagen Inc.

Noriyuki Kasahara

Christopher R Logg

James C Cicciarelli

Robert H Schiestl

Gay M Crooks

Akos Szakmary

Katrin Hacke

W French Anderson

Edward J Rebar

Jeffrey C Miller

Fyodor Urnov

Michael C Holmes

David Paschon

Lei Zhang

Gregory J Cost

Josee Laganiere

Harris Soifer

Collin Higo

Kohnosuke Mitani

Douglas J Jolly

Harry E Gruber

Amy Lin

Zea Borok

Edward Crandall

Amy H Lin

Christopher R c/oUCLA Geffen School Of Med Logg

French W Anderson

A method of radiation-free hematopoietic stem cell (HSC) transplantation comprises administering to a mammalian subject one or two doses of 2 to 10 mg/kg body weight of a purine base analog, such as 6TG as a pre-conditioning step. The method further comprises engrafting into the subject hypoxanthine-guanine phosphoribosyltransferase (HPRT)-deficient donor HSCs within 48 to 72 hours of the pre-conditioning step; and administering to the subject about 1 to 5 mg/kg of the purine base analog every two to four days for two to eight weeks following the engrafting step. The method is performed in the absence of pre-conditioning via radiation. The subject is therefore not treated with myeloablative radiation in preparation for transplantation, and thus the subject is free of myeloablative radiation-induced toxicity.

Method for chemoselection

Methods and kits are provided for determining of immunoglobulin isotypes and subclasses in a subject. In general the subject is a human who is a transplant candidate recipient or recipient, has allergies, or has an autoimmune disease. The method involves analyzing a sample of a body fluid of a transplant candidate or recipient, allergy patient or autoimmune disease sufferer and correlating the relative amounts of each immunoglobulin isotype and subtype, such that the distribution of isotypes and subtypes is an indication of success of the transplant in the candidate and recipient or the prognosis of the autoimmune disease.

Methods and kits for screening transplant recipients and candidates

This disclosure provides a retroviral replicating vector for gene delivery comprising a therapeutic cassette containing at least one mini-promoter linked to a gene to be expressed.

Retroviral vector with mini-promoter cassette

Virus vectors wherein each of the virus vectors expresses a sequence targeting a consensus conserved nucleic acid sequence, which when expressed in cells, functions as a modulator for nucleic acid encoding a domain.

Vectors and methods for long-term immune evasion to prolong transplant viability

A kit for altering allogeneic human cells for a human recipient where the kit includes a set of lentivirus vectors where each of the lentivirus vectors expresses a sequence targeting a consensus conserved nucleic acid sequence, which when expressed in cells, functions as a negative modulator for nucleic acid encoding a domain having a mismatch in an HLA protein and where the set of lentivirus vectors includes individual lentivirus vectors that correspond to individual HLA mismatches for a set of HLA mismatches that consist of HLA Class I mismatches and at least one HLA Class II mismatch and where the kit is for treatment of human cells by an appropriate subset of the set of lentivirus vectors based at least in part on a determined subset of the set of HLA mismatches between a human donor and a human recipient or between human cells and a human recipient.

Method for combined conditioning and chemoselection in a single cycle

A method can include making one or more compositions for altering allogeneic cells of a human donor for a human recipient by identifying at least one mismatch in an HLA protein between the human donor and the human recipient; determining a consensus conserved nucleic acid sequence among nucleic acid sequences encoding a domain having the mismatch or among domains having a plurality of mismatches; and forming at least one of the one or more compositions by constructing a virus vector for expressing a sequence targeting the consensus conserved nucleic acid sequence, which when expressed in cells functions as a negative modulator for nucleic acid encoding the domain having the mismatch or the domains having the plurality of mismatches.

Vectors and methods for long-term immune evasion to prolong tranplant viability

Nucleases and methods of using these nucleases for modification of an HPRT locus and for increasing the frequency of gene modification at a targeted locus and clones and for generating animals.

Methods and compositions for modification of the HPRT locus

[object Object]

A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.

Gene delivery system and methods of use

Gene delivery system and method of use

Growing community of inventors

Los Angeles, CA, United States of America

Noriyuki Kasahara