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.

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.

Laoharawee K, DeKelver RC, Podetz-Pedersen KM, Rohde M, Sproul S, Nguyen HO, Nguyen T, St Martin SJ, Ou L, Tom S, Radeke R, Meyer KE, Holmes MC, Whitley CB, Wechsler T, McIvor RS.

Mol Ther. 2018 Apr 4;26(4):1127-1136. doi: 10.1016/j.ymthe.2018.03.002. Epub 2018 Mar 10.

Categories: Genome Editing, Inherited Metabolic Diseases

NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease.

Mastaglio S, Genovese P, Magnani Z, Ruggiero E, Landoni E, Camisa B, Schiroli G, Provasi E, Lombardo A, Reik A, Cieri N, Rocchi M, Oliveira G, Escobar G, Casucci M, Gentner B, Spinelli A, Mondino A, Bondanza A, Vago L, Ponzoni M, Ciceri F, Holmes MC, Naldini L, Bonini C.

Blood. 2017 Aug 3;130(5):606-618. doi: 10.1182/blood-2016-08-732636. Epub 2017 Jun 21.

Categories: Cell Therapy, Oncology

Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells.

Chang KH, Smith SE, Sullivan T, Chen K, Zhou Q, West JA, Liu M, Liu Y, Vieira BF, Sun C, Hong VP, Zhang M, Yang X, Reik A, Urnov FD, Rebar EJ, Holmes MC, Danos O, Jiang H, Tan S.

Mol Ther Methods Clin Dev. 2017 Jan 11;4:137-148. doi: 10.1016/j.omtm.2016.12.009. eCollection 2017 Mar 17.

Categories: Cell Therapy, Hematology

Engineering hematopoietic stem cells toward a functional cure of human immunodeficiency virus infection.

Wang J, Holmes MC.

Cytotherapy. 2016 Nov;18(11):1370-1381. doi: 10.1016/j.jcyt.2016.07.007. Review.

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

State of the art: gene therapy of haemophilia.

Spencer HT, Riley BE, Doering CB.

Haemophilia. 2016 Jul;22 Suppl 5:66-71. doi: 10.1111/hae.13011. Review.

Categories: Gene Therapy, Hematology

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates.

Peterson CW, Wang J, Norman KK, Norgaard ZK, Humbert O, Tse CK, Yan JJ, Trimble RG, Shivak DA, Rebar EJ, Gregory PD, Holmes MC, Kiem HP.

Blood. 2016 May 19;127(20):2416-26. doi: 10.1182/blood-2015-09-672337. Epub 2016 Mar 15.

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

Genetic Targeting of the Albumin Locus to Treat Hemophilia.

Davidoff AM, Nathwani AC.

N Engl J Med. 2016 Mar 31;374(13):1288-90. doi: 10.1056/NEJMcibr1600347. 

Categories: Genome Editing, Hematology

Axonal transport of AAV9 in nonhuman primate brain.

Green F, Samaranch L, Zhang HS, Manning-Bog A, Meyer K, Forsayeth J, Bankiewicz KS.

Gene Ther. 2016 Jun;23(6):520-6. doi: 10.1038/gt.2016.24. Epub 2016 Mar 8.

Categories: Gene Therapy, Technology

Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery.

Wang J, DeClercq JJ, Hayward SB, Li PW, Shivak DA, Gregory PD, Lee G, Holmes MC.

Nucleic Acids Res. 2016 Feb 18;44(3):e30. doi: 10.1093/nar/gkv1121. Epub 2015 Nov 2.

Categories: Cell Therapy, Technology, Immunology, Oncology

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