Gene Therapy Protocols

ژن درمانی Gene Therapy

Recent Advances in Hemophilia Gene Therapy

Heterogeneous loss of function mutations at F8 and F9 genes causes X-linked recessive bleeding disorders, hemophilia A (HA) and hemophilia B (HB), respectively. HA is clinically indistinguishable from HB and accounts for more than 80% of hemophilia cases; the former affects 1/5000 and the latter 1/25000 male births worldwide. In Iran, it is estimated that around 4300 HA and 900 HB patients are ...

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 ...

Structuring the review of human genetics protocols, part III: gene therapy studies.

s understanding of the Astructure and functions of genes expands, so does the ability to manipulate them in new forms of treatment for inherited and acquired diseases. Gene therapy has been a topic of both scientific and ethical commentary for several decades. Actual experience in clinical trials is relatively recent, dating from the late 1980s. Since the first trial of gene transfer in humans ...

Gene therapy

In its purest form, gene therapy is the delivery and expression of a correct copy of a gene into cells in which the endogenous gene is nonfunctional. It can lead to cure — or significant enhancement of the quality of life — of the patient. The concept is highly attractive as it provides a therapeutic link between laboratory-based studies on the genetic basis of disease and attempts to alleviate...