sangamo 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.

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.

Wang J, Exline CM, DeClercq JJ, Llewellyn GN, Hayward SB, Li PW, Shivak DA, Surosky RT, Gregory PD, Holmes MC, Cannon PM.

Nat Biotechnol. 2015 Dec;33(12):1256-1263. doi: 10.1038/nbt.3408. Epub 2015 Nov 9.

Categories: Cell Therapy, Technology

Functional footprinting of regulatory DNA.

Vierstra J, Reik A, Chang KH, Stehling-Sun S, Zhou Y, Hinkley SJ, Paschon DE, Zhang L, Psatha N, Bendana YR, O'Neil CM, Song AH, Mich AK, Liu PQ, Lee G, Bauer DE, Holmes MC, Orkin SH, Papayannopoulou T, Stamatoyannopoulos G, Rebar EJ, Gregory PD, Urnov FD, Stamatoyannopoulos JA.

Nat Methods. 2015 Oct;12(10):927-30. doi: 10.1038/nmeth.3554. Epub 2015 Aug 31.

Categories: Cell Therapy, Technology, Hematology

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

Sharma R, Anguela XM, Doyon Y, Wechsler T, DeKelver RC, Sproul S, Paschon DE, Miller JC, Davidson RJ, Shivak D, Zhou S, Rieders J, Gregory PD, Holmes MC, Rebar EJ, High KA.

Blood. 2015 Oct 8;126(15):1777-84. doi: 10.1182/blood-2014-12-615492. Epub 2015 Aug 21.

Categories: Genome Editing, Inherited Metabolic Diseases, Hematology

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.

Categories: Cell Therapy, Infectious Disease (HIV/AIDS)

Toward eliminating HLA class I expression to generate universal cells from allogeneic donors.

Torikai H, Reik A, Soldner F, Warren EH, Yuen C, Zhou Y, Crossland DL, Huls H, Littman N, Zhang Z, Tykodi SS, Kebriaei P, Lee DA, Miller JC, Rebar EJ, Holmes MC, Jaenisch R, Champlin RE, Gregory PD, Cooper LJ.

Blood. 2013 Aug 22;122(8):1341-9. doi: 10.1182/blood-2013-03-478255. Epub 2013 Jun 5.

Categories: Cell Therapy

A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR.

Torikai H, Reik A, Liu PQ, Zhou Y, Zhang L, Maiti S, Huls H, Miller JC, Kebriaei P, Rabinovich B, Lee DA, Champlin RE, Bonini C, Naldini L, Rebar EJ, Gregory PD, Holmes MC, Cooper LJ.

Blood. 2012 Jun 14;119(24):5697-705. doi: 10.1182/blood-2012-01-405365. Epub 2012 Apr 24.

Categories: Cell Therapy, Oncology

In vivo genome editing restores haemostasis in a mouse model of haemophilia.

Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L, Murphy SL, Finn JD, Khazi FR, Zhou S, Paschon DE, Rebar EJ, Bushman FD, Gregory PD, Holmes MC, High KA.

Nature. 2011 Jun 26;475(7355):217-21. doi: 10.1038/nature10177.

Categories: Genome Editing, Hematology

Genome editing with engineered zinc finger nucleases.

Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD.

Nat Rev Genet. 2010 Sep;11(9):636-46. doi: 10.1038/nrg2842. Review.

Categories: Genome Editing, Technology

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. Epub 2010 Jul 2.

Categories: Cell Therapy, Infectious Disease (HIV/AIDS)

An improved zinc-finger nuclease architecture for highly specific genome editing.

Miller JC, Holmes MC, Wang J, Guschin DY, Lee YL, Rupniewski I, Beausejour CM, Waite AJ, Wang NS, Kim KA, Gregory PD, Pabo CO, Rebar EJ.

Nat Biotechnol. 2007 Jul;25(7):778-85. Epub 2007 Jul 1.

Categories: Genome Editing, Technology