Generation of Knockout Rats with X-Linked Severe Combined Immunodeficiency (X-SCID) Using Zinc-Finger Nucleases
نویسندگان
چکیده
منابع مشابه
Generation of Knockout Rats with X-Linked Severe Combined Immunodeficiency (X-SCID) Using Zinc-Finger Nucleases
BACKGROUND Although the rat is extensively used as a laboratory model, the inability to utilize germ line-competent rat embryonic stem (ES) cells has been a major drawback for studies that aim to elucidate gene functions. Recently, zinc-finger nucleases (ZFNs) were successfully used to create genome-specific double-stranded breaks and thereby induce targeted gene mutations in a wide variety of ...
متن کاملKnockout rats via embryo microinjection of zinc-finger nucleases.
The toolbox of rat genetics currently lacks the ability to introduce site-directed, heritable mutations into the genome to create knockout animals. By using engineered zinc-finger nucleases (ZFNs) designed to target an integrated reporter and two endogenous rat genes, Immunoglobulin M (IgM) and Rab38, we demonstrate that a single injection of DNA or messenger RNA encoding ZFNs into the one-cell...
متن کاملEfficacy of gene therapy for X-linked severe combined immunodeficiency.
BACKGROUND The outcomes of gene therapy to correct congenital immunodeficiencies are unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked severe combined immunodeficiency (SCID-X1), which is characterized by the absence of the cytokine receptor common gamma chain. METHODS The nine patients, who lacked an HLA-identical donor, underwent ex vivo retrovirus-me...
متن کاملSafety Modality for X-linked Severe Combined Immunodeficiency Gene Therapy
Abbreviations: SCID-X1: X-linked Severe Combined Immunodeficiency; γc: Cytokine Receptor Common Gamma Chain; HSC: Hematopoietic Stem Cells; ab: Antibody; wt: Wild-type; NK cell: Natural Killer Cell; HLA: Human Leukocyte Antigen; SIN: Selfinactivating; HSV-TK: Thymidine kinase of Herpes Simplex Virus; ER: Endoplasmatic Reticulum; MFI: Mean Fluorescence Intensity; CDC: Complement-dependent Cytoto...
متن کاملBone marrow transplantation for canine X-linked severe combined immunodeficiency.
Canine X-linked severe combined immunodeficiency (XSCID) is due to mutations in the common gamma chain which is a subunit of the receptors of IL-2, IL-4, IL-7, IL-9 and IL-15. Bone marrow transplantation (BMT) of human XSCID patients without pretransplant conditioning (cytoablation) results in engraftment of donor T-cells and reconstitution of T-cell function but engraftment of few, if any, don...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: PLoS ONE
سال: 2010
ISSN: 1932-6203
DOI: 10.1371/journal.pone.0008870