Correction: Targeted gene correction in the mdx mouse using short DNA fragments: towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle
نویسندگان
چکیده
A Correction to this paper has been published: https://doi.org/10.1038/s41434-020-00217-7
منابع مشابه
Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells.
Duchenne muscular dystrophy (DMD) is an X-linked, lethal disease caused by mutations of the dystrophin gene. No effective therapy is available, but dystrophin gene transfer to skeletal muscle has been proposed as a treatment for DMD. We have developed a strategy for efficient in vivo gene transfer of dystrophin cDNA into regenerating skeletal muscle. Retroviral producer cells, which release a v...
متن کاملComparative analysis of the Gene expression profile of Chemokine Receptors between Adipose-derived and Bone marrow-derived Mesenchymal Stem Cells
Introduction: Mesenchymal stem cells (MSCs) hold great promise in the field of regenerative medicine.Although originally isolated from bone marrow, MSCs have since been obtained from a variety of adult and neonatal tissues including the adipose tissue. Stemness and multipotential features of Mesenchymal Stem Cells (MSC) has been highlighted in many studies but there are many dark aspects in ex...
متن کاملFunctional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin.
Duchenne muscular dystrophy is a lethal X-linked recessive disorder caused by mutations in the dystrophin gene. Delivery of functionally effective levels of dystrophin to immunocompetent, adult mdx (dystrophin-deficient) mice has been challenging because of the size of the gene, immune responses against viral vectors, and inefficient infection of mature muscle. Here we show that high titer stoc...
متن کاملRetrovirally mediated correction of bone marrow-derived mesenchymal stem cells from patients with mucopolysaccharidosis type I.
We have investigated the utility of bone marrow-derived mesenchymal stem cells (MSCs) as targets for gene therapy of the autosomal recessive disorder mucopolysaccharidosis type IH (MPS-IH, Hurler syndrome). Cultures of MSCs were initially exposed to a green fluorescent protein-expressing retrovirus. Green fluorescent protein-positive cells maintained their proliferative and differentiation capa...
متن کاملThe Regenerative Effect of Bone Marrow-Derived Stem Cells in Spermatogenesis of Infertile Hamster
BACKGROUND Infertility is a serious social problem in advanced nations, with male factor in half of all cases of infertility. This study was conducted to determine the regenerative effect of bone marrow-derived stem cells in spermatogenesis of infertile hamster. METHODS Twelve adult male hamsters were equally divided into azoospermic and control groups. Busulfan was intraperitoneally used fo...
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: Gene Therapy
سال: 2021
ISSN: ['0969-7128', '1476-5462']
DOI: https://doi.org/10.1038/s41434-020-00217-7