Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease.

Autor: Zeitler B; Sangamo Therapeutics, Inc., Richmond, CA, USA. bzeitler@sangamo.com., Froelich S; Sangamo Therapeutics, Inc., Richmond, CA, USA., Marlen K; Sangamo Therapeutics, Inc., Richmond, CA, USA., Shivak DA; Sangamo Therapeutics, Inc., Richmond, CA, USA., Yu Q; Sangamo Therapeutics, Inc., Richmond, CA, USA., Li D; Sangamo Therapeutics, Inc., Richmond, CA, USA., Pearl JR; Sangamo Therapeutics, Inc., Richmond, CA, USA., Miller JC; Sangamo Therapeutics, Inc., Richmond, CA, USA., Zhang L; Sangamo Therapeutics, Inc., Richmond, CA, USA., Paschon DE; Sangamo Therapeutics, Inc., Richmond, CA, USA., Hinkley SJ; Sangamo Therapeutics, Inc., Richmond, CA, USA., Ankoudinova I; Sangamo Therapeutics, Inc., Richmond, CA, USA., Lam S; Sangamo Therapeutics, Inc., Richmond, CA, USA., Guschin D; Sangamo Therapeutics, Inc., Richmond, CA, USA.; Laboratory of Intracellular Signalling, Moscow Institute of Physics and Technology, Dolgoprudnyi, Russian Federation., Kopan L; Sangamo Therapeutics, Inc., Richmond, CA, USA., Cherone JM; Sangamo Therapeutics, Inc., Richmond, CA, USA., Nguyen HB; Sangamo Therapeutics, Inc., Richmond, CA, USA., Qiao G; Sangamo Therapeutics, Inc., Richmond, CA, USA., Ataei Y; Sangamo Therapeutics, Inc., Richmond, CA, USA., Mendel MC; Sangamo Therapeutics, Inc., Richmond, CA, USA., Amora R; Sangamo Therapeutics, Inc., Richmond, CA, USA., Surosky R; Sangamo Therapeutics, Inc., Richmond, CA, USA., Laganiere J; Sangamo Therapeutics, Inc., Richmond, CA, USA.; Medical Affairs and Innovation, Hema-Quebec, Quebec City, Quebec, Canada., Vu BJ; Sangamo Therapeutics, Inc., Richmond, CA, USA., Narayanan A; Sangamo Therapeutics, Inc., Richmond, CA, USA., Sedaghat Y; Evotec AG, Hamburg, Germany., Tillack K; Evotec AG, Hamburg, Germany., Thiede C; Evotec AG, Hamburg, Germany., Gärtner A; Evotec AG, Hamburg, Germany., Kwak S; CHDI Management/CHDI Foundation, Los Angeles, CA, USA., Bard J; CHDI Management/CHDI Foundation, Los Angeles, CA, USA., Mrzljak L; CHDI Management/CHDI Foundation, Los Angeles, CA, USA., Park L; CHDI Management/CHDI Foundation, Los Angeles, CA, USA., Heikkinen T; Charles River Discovery Services, Kuopio, Finland., Lehtimäki KK; Charles River Discovery Services, Kuopio, Finland., Svedberg MM; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden., Häggkvist J; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden., Tari L; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden., Tóth M; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden., Varrone A; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden., Halldin C; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden., Kudwa AE; Psychogenics, Inc., Paramus, NJ, USA., Ramboz S; Psychogenics, Inc., Paramus, NJ, USA., Day M; Department of Physiology, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA., Kondapalli J; Department of Physiology, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA., Surmeier DJ; Department of Physiology, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA., Urnov FD; Sangamo Therapeutics, Inc., Richmond, CA, USA.; Innovative Genomics Institute, Berkeley, CA, USA., Gregory PD; Sangamo Therapeutics, Inc., Richmond, CA, USA., Rebar EJ; Sangamo Therapeutics, Inc., Richmond, CA, USA., Muñoz-Sanjuán I; CHDI Management/CHDI Foundation, Los Angeles, CA, USA. ignacio.munoz@chdifoundation.org., Zhang HS; Sangamo Therapeutics, Inc., Richmond, CA, USA.; Applied StemCell, Inc., Milpitas, CA, USA.

Publikováno v: Nature medicine [Nat Med] 2019 Jul; Vol. 25 (7), pp. 1131-1142. Date of Electronic Publication: 2019 Jul 01.

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.

Autor: Hoban MD; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Cost GJ; Sangamo BioSciences Inc., Richmond, CA;, Mendel MC; Sangamo BioSciences Inc., Richmond, CA;, Romero Z; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Kaufman ML; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Joglekar AV; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Ho M; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Lumaquin D; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Gray D; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Lill GR; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Cooper AR; Molecular Biology Interdepartmental PhD Program, University of California, Los Angeles, Los Angeles, CA;, Urbinati F; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Senadheera S; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Zhu A; Sangamo BioSciences Inc., Richmond, CA;, Liu PQ; Sangamo BioSciences Inc., Richmond, CA;, Paschon DE; Sangamo BioSciences Inc., Richmond, CA;, Zhang L; Sangamo BioSciences Inc., Richmond, CA;, Rebar EJ; Sangamo BioSciences Inc., Richmond, CA;, Wilber A; Department of Medical Microbiology, Immunology and Cell Biology, Southern Illinois University School of Medicine, Springfield, IL;, Wang X; Department of General Internal Medicine and Health Services Research, University of California, Los Angeles, Los Angeles, CA; and., Gregory PD; Sangamo BioSciences Inc., Richmond, CA;, Holmes MC; Sangamo BioSciences Inc., Richmond, CA;, Reik A; Sangamo BioSciences Inc., Richmond, CA;, Hollis RP; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Kohn DB; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA.

Publikováno v: Blood [Blood] 2015 Apr 23; Vol. 125 (17), pp. 2597-604. Date of Electronic Publication: 2015 Mar 02.

Combining old and new concepts in targeting telomerase for cancer therapy: transient, immediate, complete and combinatory attack (TICCA).

Autor: Ali, Jaber Haj^1,2 (AUTHOR), Walter, Michael² (AUTHOR) michael.walter@med.uni-rostock.de

Publikováno v: Cancer Cell International. 9/26/2023, Vol. 23 Issue 1, p1-24. 24p.

hESC-derived striatal progenitors grafted into a Huntington's disease rat model support long-term functional motor recovery by differentiating, self-organizing and connecting into the lesioned striatum.

Autor: Schellino, Roberta^1,2 (AUTHOR) roberta.schellino@unito.it, Besusso, Dario^3,4 (AUTHOR), Parolisi, Roberta^1,2 (AUTHOR), Gómez-González, Gabriela B.^1,2 (AUTHOR), Dallere, Sveva^1,2 (AUTHOR), Scaramuzza, Linda^3,4 (AUTHOR), Ribodino, Marta^1,2 (AUTHOR), Campus, Ilaria^3,4 (AUTHOR), Conforti, Paola^3,4 (AUTHOR), Parmar, Malin⁵ (AUTHOR), Boido, Marina^1,2 (AUTHOR), Cattaneo, Elena^3,4 (AUTHOR), Buffo, Annalisa^1,2 (AUTHOR) annalisa.buffo@unito.it

Publikováno v: Stem Cell Research & Therapy. 7/28/2023, Vol. 14 Issue 1, p1-21. 21p.

Isogenic human cell lines for drug discovery: regulation of target gene expression by engineered zinc-finger protein transcription factors.

Autor: Liu PQ; Sangamo BioSciences, Inc., Richmond, CA 94804, USA., Tan S, Mendel MC, Murrills RJ, Bhat BM, Schlag B, Samuel R, Matteo JJ, de la Rosa R, Howes K, Reik A, Case CC, Bex FJ, Young K, Gregory PD

Publikováno v: Journal of biomolecular screening [J Biomol Screen] 2005 Jun; Vol. 10 (4), pp. 304-13.