Recent evidence demonstrating the feasibility of using novel CRISPR/Cas9 gene editing technology to make targeted changes in the DNA of human embryos is forcing researchers, clinicians, and ethicists to revisit the highly ...
May 1, 2015
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Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response. ...
Mar 12, 2015
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Excessive and often lethal blood levels of bilirubin can result from mutations in a single gene that are the cause of the metabolic disease known as Crigler-Najjar syndrome type 1 (CNS1). A new gene therapy approach to correcting ...
Oct 9, 2014
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In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. Developments in this rapidly advancing field have already ...
Jul 17, 2014
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Scientists working to make gene therapy a reality have solved a major hurdle: how to bypass a blood stem cell's natural defenses and efficiently insert disease-fighting genes into the cell's genome.
Jun 26, 2014
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To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords ...
May 22, 2014
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A targeted gene silencing strategy blocks production of the dysfunctional huntingtin (Htt) protein, the cause of Huntington's disease, a fatal, inherited neurodegenerative disorder. The effectiveness of this RNA interference ...
May 21, 2014
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Several young children suffering from a severe degenerative genetic disease received injections of therapeutic genes packaged within a noninfectious viral delivery vector. Safety, tolerability, and efficacy results from this ...
Mar 11, 2014
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Packaging replacement genes in viruses is an effective method to deliver them to target tissues, but the human body mounts an immune response against the virus. The systemic and local immune reactions induced by an adeno-associated ...
Jan 8, 2014
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Cancer immunotherapy can successfully use the body's own immune system to kill tumor cells. But some current approaches to stimulate an antitumor immune response are short-lived, with limited clinical effectiveness. A new ...
Oct 9, 2013
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