human gene therapy Latest News

A novel therapeutic approach called exon skipping involves bypassing a disease-causing mutation in a gene to restore normal gene expression and protein production.

Targeted T-cells can seek out and destroy tumor cells that carry specific antigen markers. Two novel anti-tumor therapies that take advantage of this T-cell response are described in articles published in Human Gene Therapy.

Human Gene Therapy is expanding its editorial coverage with the launch of Human Gene Therapy Clinical Development early next year.

The body’s natural reaction to reject replacement proteins represents a major obstacle to the successful use of gene therapy to cure a range of life-threatening diseases.

A novel approach to gene therapy that instructs a person’s own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders.

Gene therapy to replace the protein missing in Pompe disease can be effective if the patient’s immune system does not react against the therapy.

Gene therapy strategies to prevent and treat inherited diseases of the retina that can cause blindness have progressed rapidly. Positive results in animal models of human retinal disease continue to emerge, as reported in Human Gene Therapy.

The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology.

A recent article in the New England Journal of Medicine showed that Factor IX gene therapy in patients with hemophilia B was able to convert severe hemophilia to moderate or even mild disease

New Rochelle, NY, September 13, 2011–Mary Ann Liebert, Inc. announces the launch of a journal expansion, Human Gene Therapy Methods, to complement the flagship publication Human Gene Therapy. HGT Methods.

Sophisticated genetic tools and techniques for achieving targeted gene delivery and high gene expression levels in bone marrow will drive the successful application of gene therapy to treat a broad range of diseases.

Novel tools and methods for delivering therapeutic genes to cells in the central nervous system hold great promise for the development of new treatments to combat incurable neurologic diseases.

Many gene therapy strategies designed to deliver a normal copy of a gene to cells carrying a disease-causing genetic mutation rely on a modified virus to transfer the gene product into affected tissues.

Disorders of the eye are excellent targets for gene therapy because the ocular environment is readily accessible, relatively easy to monitor, and sequestered from the rest of the body

An innovative genetic strategy for rendering T-cells resistant to HIV infection without affecting their normal growth and activity is described in a paper published in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc.

Give caffeine to cells engineered to produce viruses used for gene therapy and the cells can generate 3- to 8-times more virus, according to a paper published in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc.

A new, simplified method for producing large amounts of viral vector cassettes capable of shuttling genes into host cells will help advance the promising field of gene therapy as applications move into large animal studies and human clinical trials.

The introduction of a new, fully characterized viral vector for use as reference material to help standardize gene therapy protocols in research applications and human clinical trials is described in an article in Human Gene Therapy

The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit.

Improved delivery methods and better testing systems are needed to advance promising gene therapy strategies for treating prostate cancer, according to a series of review articles in Human Gene Therapy

All three patients receiving intraocular adeno-associated virus-based gene therapy to treat Leber congenital amaurosis (LCA) an incurable genetic disorder resulting in blindness had improved vision according to a paper published in Human Gene Therapy

Many recent reports documenting the therapeutic efficacy of short interfering RNAs (siRNAs) in animal models of human disease may actually be describing non-specific therapeutic effects related to the ability of siRNA to activate an immune response.

Gene therapy to replace the faulty CLN2 gene, which causes a neurodegenerative disease that is fatal by age 8-12 years, was able to slow significantly the rate of neurologic decline in treated children.

RNAi represents an innovative new strategy for using small RNA molecules to silence specific genes associated with disease processes, and a series of review articles describing applications of RNAi and microRNAs will begin in Human Gene Therapy.