Generation of transgenic mice using lentiviral vectors: a novel preclinical assessment of lentiviral vectors for gene therapy

نویسندگان
چکیده

برای دانلود باید عضویت طلایی داشته باشید

برای دانلود متن کامل این مقاله و بیش از 32 میلیون مقاله دیگر ابتدا ثبت نام کنید

اگر عضو سایت هستید لطفا وارد حساب کاربری خود شوید

منابع مشابه

Beta thalassemia gene therapy using lentiviral vectors

Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising ...

متن کامل

Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors.

Fanconi anemia (FA) is an inherited cancer susceptibility syndrome caused by mutations in a DNA repair pathway including at least 6 genes (FANCA, FANCC, FANCD2, FANCE, FANCF, and FANCG). The clinical course of the disease is dominated by progressive, life-threatening bone marrow failure and high incidence of acute myelogenous leukemia and solid tumors. Allogeneic bone marrow transplantation (BM...

متن کامل

Lentiviral vectors and gene therapy.

Gene therapy is a novel method under investigation for the treatment of genetic, metabolic and neurologic diseases, cancer and AIDS. The primary goal of gene therapy is to deliver a specific gene to a pre-determined target cell, and to direct expression of such a gene in a manner which will result in a therapeutic effect. Retroviral vectors have the ability to integrate in the host cell DNA irr...

متن کامل

Lentiviral Vectors: Not Just For Gene Therapy

Lentiviral vectors (LVs) are viral-based gene delivery systems that can stably deliver genes or RNAi into primary cells or cell lines with up to 100% efficiency. LVs bind to target cells using an envelope protein which allows for release of the LV RNA containing the gene or gene silencing sequence into the cell. The LV’s RNA is then converted into DNA using an enzyme called reverse transcriptas...

متن کامل

Designing Lentiviral Gene Vectors

Gene therapy relies on the delivery of therapeutic genes into patients’ cells. The microdevices used to reach the cells and to transfer the gene payload are called gene vectors. Viral packaging machinery is often utilized to generate the particles transporting the cargo genes. Lentiviruses, a subgroup of retroviruses, are highly suitable for remodeling into gene transfer vectors because they of...

متن کامل

ذخیره 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: Molecular Therapy

سال: 2003

ISSN: 1525-0016

DOI: 10.1016/s1525-0016(03)00240-5