In vivo correction of ZAP-70 immunodeficiency by intrathymic gene transfer
نویسندگان
چکیده
منابع مشابه
In vivo correction of ZAP-70 immunodeficiency by intrathymic gene transfer.
SCID patients have been successfully treated by administration of ex vivo gene-corrected stem cells. However, despite its proven efficacy, such treatment carries specific risks and difficulties. We hypothesized that some of these drawbacks may be overcome by in situ gene correction of T lymphoid progenitors in the thymus. Indeed, in vivo intrathymic transfer of a gene that provides a selective ...
متن کاملIntrathymic administration of hematopoietic progenitor cells enhances T cell reconstitution in ZAP-70 severe combined immunodeficiency.
Patients with severe combined immunodeficiency (SCID) present with opportunistic infections that are almost universally fatal in infancy. The mainstay treatment for these patients is allogeneic hematopoietic stem cell (HSC) transplantation, but sustained polyclonal T cell reconstitution is too often unsatisfactory. Although transplantation is conventionally performed by i.v. administration of H...
متن کاملZAP-70 Restoration in Mice by In Vivo Thymic Electroporation
Viral and non-viral vectors have been developed for gene therapy, but their use is associated with unresolved problems of efficacy and safety. Efficient and safe methods of DNA delivery need to be found for medical application. Here we report a new monopolar system of non-viral electro-gene transfer into the thymus in vivo that consists of the local application of electrical pulses after the in...
متن کاملReconstitution of T cell receptor signaling in ZAP-70-deficient cells by retroviral transduction of the ZAP-70 gene
A variant of severe combined immunodeficiency syndrome (SCID) with a selective inability to produce CD8 single positive T cells and a signal transduction defect in peripheral CD4+ cells has recently been shown to be the result of mutations in the ZAP-70 gene. T cell receptor (TCR) signaling requires the association of the ZAP-70 protein tyrosine kinase with the TCR complex. Human T cell leukemi...
متن کاملpragmatic transfer in compliment responses and chastisements by iranian learners of english
فرهنگ های گوناگون دریافت ها و تعبیر های مختلفی از تناسب و ادب دارند. بنابراین، ارتباط میان فرهنگی خطرات ذاتی خطای ارتباطی را نشان می دهد. این پژوهش به منظور تعیین وقوع انتقال منظورشناختی زبان در زبان بینابینی زبان آموزان ایرانی، اختلاف منظورشناختی زبان را بین فارسی و انگلیسی در کنش های کلامی پاسخ تحسین و انتقاد بررسی می کند.داده ها از میان 33 آمریکایی انگلیسی زبان، 85 زبان آموز ایرانی در سطوح م...
15 صفحه اولذخیره 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: Journal of Clinical Investigation
سال: 2005
ISSN: 0021-9738
DOI: 10.1172/jci23966