Publications

Presentations + Publications

Our robust collection of publications, authored by our own scientists, collaborators and others, contains original research articles and review articles that are relevant to our ZFP technology and its many applications.

Highly Efficient and Specific Multiplexed Gene Editing in T Cells Using Enhanced Zinc-Finger Nucleases (ZFNs) Enables Strategic Engineering of Allogeneic T Cell Immunotherapies

Sumiti Jain, Ph.D.

Enhancing ZFN Expression Construct and Nuclease Activity Leads to Improvement of In Vivo Genome Editing Platform

Russell DeKelver, Ph.D.

Designed Zinc Finger Protein Transcription Factors for Single-Gene Regulation Throughout the Central Nervous System

Bryan Zeitler, Ph.D.

Global and Tunable Suppression of Zinc Finger Nuclease and ZFP-Transcription Factor Off-target Activity via Discrete Framework Substitutions

Edward Rebar, Ph.D.

AAV-mediated expression of human α-galactosidase A gene in hepatocytes of a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction

Marshall Huston, Ph.D.

ZFN-mediated expression of the human α-galactosidase A gene in hepatocytes of a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction

Marshall Huston, Ph.D.

Non-viral Delivery of ZFN mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets

Anthony Conway, Ph.D.

ZFN Genome Editing Technology

Sangamo Therapeutics

CNS Gene Regulation Platform

Sangamo Therapeutics

Cell Therapies for Immuno-Oncology

Sangamo Therapeutics

Dual Knock-Out of Endogenous T-Cell Receptor and HLA-Class I using Zinc Finger Nucleases with Site-Specific Insertion of a CD19 CAR: Implications for Allogeneic T Cell Therapy

Sumiti Jain, Ph.D.

Update on Phase 1/2 Clinical Trials for MPS I and MPS II Using ZFN-mediated in vivo Genome Editing

Paul Harmatz, M.D.

Engineering T-cells for Cancer Therapeutics

Gary Lee, Ph.D.

New Zinc Finger Nuclease Architectures for Precision Genome Engineering

Edward Rebar, Ph.D.

Sustained Tau Reduction via Zinc Finger Protein Transcription Factors as a Potential Next-Generation Therapy for Alzheimer’s Disease and Other Tauopathies

Bryan Zeitler, Ph.D.

New Zinc Finger Nuclease Architectures for Highly Efficient Genome Engineering in Primary Cells at Large Scale with No Detectable Off-Target Effects

Edward Rebar, Ph.D.

ZFN-Mediated In Vivo Genome Editing Results in Phenotypic Correction in MPS I and MPS II Mouse Models

Russell DeKelver, Ph.D.

Liver-based expression of the human alpha-galactosidase A gene (GLA) in a murine Fabry model results in continuous high levels of enzyme activity and effective substrate reduction

Thomas Wechsler, Ph.D.

Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells.

DiGiusto DL, Cannon PM, Holmes MC, Li L, Rao A, Wang J, Lee G, Gregory PD, Kim KA, Hayward SB, Meyer K, Exline C, Lopez E, Henley J, Gonzalez N, Bedell V, Stan R, Zaia JA.
Mol Ther Methods Clin Dev. 2016 Nov 9;3:16067

Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV.

Tebas P, Stein D, Tang WW, Frank I, Wang SQ, Lee G, Spratt SK, Surosky RT, Giedlin MA, Nichol G, Holmes MC, Gregory PD, Ando DG, Kalos M, Collman RG, Binder-Scholl G, Plesa G, Hwang WT, Levine BL, June CH.
N Engl J Med. 2014 Mar 6;370(10):901-10. doi: 10.1056/NEJMoa1300662.

Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases.

Li L, Krymskaya L, Wang J, Henley J, Rao A, Cao LF, Tran CA, Torres-Coronado M, Gardner A, Gonzalez N, Kim K, Liu PQ, Hofer U, Lopez E, Gregory PD, Liu Q, Holmes MC, Cannon PM, Zaia JA, DiGiusto DL.
Mol Ther. 2013 Jun;21(6):1259-69. doi: 10.1038/mt.2013.65

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo.

Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC, Cannon PM.
Nat Biotechnol. 2010 Aug;28(8):839-47. doi: 10.1038/nbt.1663

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Rajiv Sharma, Xavier M. Anguela, Yannick Doyon, Thomas Wechsler, Russell C. DeKelver, Scott Sproul, David E. Paschon, Jeffrey C. Miller, Robert J. Davidson, David Shivak, Shangzhen Zhou, Julianne Rieders, Philip D. Gregory, Michael C. Holmes, Edward J. Rebar, and Katherine A. High
BLOOD, 8 OCTOBER 2015 x VOLUME 126, NUMBER 15

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Rajiv Sharma, Xavier M. Anguela, Yannick Doyon, Thomas Wechsler, Russell C. DeKelver, Scott Sproul, David E. Paschon, Jeffrey C. Miller, Robert J. Davidson, David Shivak, Shangzhen Zhou, Julianne Rieders, Philip D. Gregory, Michael C. Holmes, Edward J. Rebar and Katherine A. High
BLOOD, 8 OCTOBER 2015 x VOLUME 126, NUMBER 15