Gene Replacement Therapy by Gene Transfer Into HSCs Reference Disease Gene Defect
نویسنده
چکیده
Information about subscriptions and ASH membership may be found online at: reserved.
منابع مشابه
Designing an Engineered Construct Gene Sensitive to Carbohydrate In-vitro and Candidate for Human Insulin Gene Therapy In-vivo
Background and Aim: Diabetes is a common disorder worldwide, and exhaustive efforts have been made to cure this disease. Gene therapy has considered as a potential curative method that has more stability in comparison with the other pharmaceutical methods. However, the application of gene therapy as a definitive treatment demands further investigation. This study aim is to prepare a suitable hi...
متن کاملDesigning an Engineered Construct Gene Sensitive to Carbohydrate In-vitro and Candidate for Human Insulin Gene Therapy In-vivo
Background and Aim: Diabetes is a common disorder worldwide, and exhaustive efforts have been made to cure this disease. Gene therapy has considered as a potential curative method that has more stability in comparison with the other pharmaceutical methods. However, the application of gene therapy as a definitive treatment demands further investigation. This study aim is to prepare a suitable hi...
متن کاملP96: Progress in the Treatment of Alzheimer’s Disease by Gene Therapy
Alzheimer’s disease (AD) is a progressive neurological disorder characterized by the aggregation of two proteins, amyloid-b and hyper phosphorylated tau, and by neuronal and synaptic loss. The progress of gene-modified cells and stem cells is a particularly promising therapeutic method for AD. Gene-Modified Cell-Based Therapy for AD prior to transplantation can be beneficial for increasin...
متن کاملCorrection of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.
Hematopoietic stem cell (HSC) gene therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID) has shown limited clinical efficacy because of the small proportion of engrafted genetically corrected HSCs. We describe an improved protocol for gene transfer into HSCs associated with nonmyeloablative conditioning. This protocol was used in two patients for whom enzyme re...
متن کاملP-111: An Attempt to Facilitate the Production of Transgenic Mouse As A Model for Gene Therapy of Gaucher Disease
Background: Gaucher disease is an autosomal recessive inherited lysosomal storage disorder that affects many of the body's organs and tissues by defective function of the catabolic enzyme β-glucocerebrosidase. Gene therapy is one of the efficient ways for treatment of this disease. Due to the lack of appropriate animal models, in the field of gene therapy little progress has been done.Mate...
متن کامل