CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease.

Autor: De Ravin SS; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA. sderavin@niaid.nih.gov hmalech@nih.gov., Li L; MaxCyte Inc., Gaithersburg, MD 20878, USA., Wu X; Cancer Research Technology Program, Leidos Biomedical Research Inc., Frederick, MD 21701, USA., Choi U; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Allen C; MaxCyte Inc., Gaithersburg, MD 20878, USA., Koontz S; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Lee J; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Theobald-Whiting N; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Chu J; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Garofalo M; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Sweeney C; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Kardava L; Laboratory of Immunoregulation, National Institutes of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Moir S; Laboratory of Immunoregulation, National Institutes of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Viley A; MaxCyte Inc., Gaithersburg, MD 20878, USA., Natarajan P; MaxCyte Inc., Gaithersburg, MD 20878, USA., Su L; Cancer Research Technology Program, Leidos Biomedical Research Inc., Frederick, MD 21701, USA., Kuhns D; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Zarember KA; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA., Peshwa MV; MaxCyte Inc., Gaithersburg, MD 20878, USA., Malech HL; Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA. sderavin@niaid.nih.gov hmalech@nih.gov.

Publikováno v: Science translational medicine [Sci Transl Med] 2017 Jan 11; Vol. 9 (372).

Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood Disorders.

Autor: Allen, Daniel¹ (AUTHOR) dallen413@gmail.com, Kalter, Nechama¹ (AUTHOR), Rosenberg, Michael¹ (AUTHOR), Hendel, Ayal¹ (AUTHOR) ayal.hendel@biu.ac.il

Publikováno v: Pharmaceutics. May2023, Vol. 15 Issue 5, p1329. 18p.

Developing CRISPR/Cas9-Mediated Fluorescent Reporter Human Pluripotent Stem-Cell Lines for High-Content Screening.

Autor: Vojnits, Kinga¹ (AUTHOR) vojnitsk@mcmaster.ca, Nakanishi, Mio¹ (AUTHOR), Porras, Deanna¹ (AUTHOR), Kim, Yeonjoon¹ (AUTHOR), Feng, Zhuohang¹ (AUTHOR), Golubeva, Diana¹ (AUTHOR), Bhatia, Mick¹ (AUTHOR) mbhatia@mcmaster.ca

Publikováno v: Molecules. Apr2022, Vol. 27 Issue 8, pN.PAG-N.PAG. 16p.

Engineering precision therapies: lessons and motivations from the clinic.

Autor: Xie, Mingqi^1,2,3 xiemingqi@westlake.edu.cn, Viviani, Mirta^1,2,3, Fussenegger, Martin^4,5 xiemingqi@westlake.edu.cn

Publikováno v: Synthetic Biology (23977000). 2021, Vol. 6 Issue 1, p1-21. 21p.

Successful application of next-generation sequencing for pre-natal diagnosis in a pedigree with chronic granulomatosis disease.

Autor: Peng, Fang¹ (AUTHOR), Zhong, Lili¹ (AUTHOR), Zhang, Bing¹ (AUTHOR), Zou, Runying¹ (AUTHOR), Nie, Shengdan¹ (AUTHOR), Tian, Xin¹ (AUTHOR), Deng, Shaoyang¹ (AUTHOR), He, Xiangling¹ (AUTHOR) hexiangl@163.com

Publikováno v: Experimental & Therapeutic Medicine. Apr2019, Vol. 17 Issue 4, p2931-2936. 6p.