Fragile X Syndrome Patient-Derived Neurons Developing in the Mouse Brain Show FMR1-Dependent Phenotypes.

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第一作者： Marine A,Krzisch

第一单位： Whitehead Institute for Biomedical Research, Cambridge, Massachusetts. Electronic address: mkrzisch@wi.mit.edu.

作者： Marine A,Krzisch ^[1] ; Hao,Wu ^[2] ; Bingbing,Yuan ^[3] ; Troy W,Whitfield ^[3] ; X Shawn,Liu ^[4] ; Dongdong,Fu ^[3] ; Carrie M,Garrett-Engele ^[3] ; Andrew S,Khalil ^[3] ; Tenzin,Lungjangwa ^[3] ; Jennifer,Shih ^[5] ; Aaron N,Chang ^[6] ; Stephen,Warren ^[7] ; Angela,Cacace ^[8] ; Kristin R,Andrykovich ^[3] ; Rosalie G J,Rietjens ^[3] ; Owen,Wallace ^[9] ; Mriganka,Sur ^[5] ; Bhav,Jain ^[3] ; Rudolf,Jaenisch ^[10]

作者单位： Whitehead Institute for Biomedical Research, Cambridge, Massachusetts. Electronic address: mkrzisch@wi.mit.edu. ^[1] Full Circles Therapeutics, Inc., Cambridge, Massachusetts. ^[2] Whitehead Institute for Biomedical Research, Cambridge, Massachusetts. ^[3] Department of Physiology and Cellular Biophysics, Columbia University Medical Center, New York, New York. ^[4] Picower Institute for Learning and Memory, Cambridge, Massachusetts. ^[5] Prime Medicine, Inc., Cambridge, Massachusetts. ^[6] Departments of Human Genetics, Biochemistry, and Pediatrics, Emory University School of Medicine, Atlanta, Georgia. ^[7] Arvinas, New Haven, Connecticut. ^[8] Monte Rosa Therapeutics, Boston, Massachusetts. ^[9] Whitehead Institute for Biomedical Research, Cambridge, Massachusetts; Department of Biology, Massachusetts Institute of Technology, Cambridge, Massachusetts. Electronic address: jaenisch@wi.mit.edu. ^[10]

关键词 CGG repeats CRISPR/Cas9 Engraftment FMRP Fragile X syndrome Phenotype Single-cell sequencing