New system for therapeutic gene delivery increases transgene expression up to...
Advanced engineering of a mini-intronic plasmid (MIP) system designed to carry a therapeutic gene can significantly enhance the expression of the transgene delivered using an adeno-associated viral...
View ArticleHormone may offer new contraceptive that protects ovaries from chemotherapy
A naturally occurring hormone that plays an important role in fetal development may be the basis for a new type of reversible contraceptive that can protect ovaries from the damage caused by...
View ArticleGene therapy restores hearing in deaf mice... down to a whisper
In the summer of 2015, a team at Boston Children's Hospital and Harvard Medical School reported restoring rudimentary hearing in genetically deaf mice using gene therapy. Now the Boston Children's...
View ArticleA form of RNA released from fat cells into the blood may help to regulate...
Fat cells are not simply big blobs of lipid quietly standingby in the body—instead, they send out hormones and other signaling proteins that affect many types of tissues. Scientists at Joslin Diabetes...
View ArticleGene therapy treats muscle-wasting disease in dogs
Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for...
View ArticleHuman genome editing report strikes the right balance between risks and benefits
If you recognise the words "CRISPR-mediated gene editing", then you'll know that our ability to alter DNA has recently become much more efficient, faster and cheaper.
View ArticleGene therapy used to treat Fabry disease—a world first
A team of Canadian physicians and researchers is believed to be the first in the world to have used gene therapy to treat a patient with Fabry disease, a rare inherited enzyme deficiency that can...
View ArticleDefying Mendelian genetics and 'embryo engineering'
Mendel's laws, like any laws in science, are wonderful because they make predictions possible. A woman and man both carry a recessive mutation in the same gene, and each of their children has a 25%...
View ArticleAAV gene delivery vectors and cancer—The debate continues
Overwhelming evidence from the biomedical literature shows that adeno-associated virus 2 (AAV2), a viral vector often used to deliver therapeutic genes, is not associated with cancer and, in fact, may...
View ArticleResearch asks why genetic mutation leads to decreased triglycerides in blood
Common knowledge says that genetic mutations are bad. This is true for most mutations of lipoprotein lipase (LPL), the enzyme in the blood responsible for the breakdown of lipoproteins, which allows...
View ArticleGene therapy lets a French teen dodge sickle cell disease
A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.
View ArticleNew trial for blindness rewrites the genetic code
Researchers have started a new gene therapy clinical trial to treat X-linked retinitis pigmentosa (XLRP), the most common cause of blindness in young people.
View ArticleWill AAV vectors have a role in future novel gene therapy approaches?
Recombinant adeno-associated virus (rAAV) vectors for delivering therapeutic genes have demonstrated their safety in multiple diseases and clinical settings over the years and are a proven and...
View ArticleTesting the efficacy of new gene therapies more efficiently
Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and cost-effectively in the lab for their efficacy....
View ArticleNovel gene therapy experiment offers hope for people with certain hearing...
In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins...
View ArticlePioneering stem cell gene therapy cures infants with bubble baby disease
UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be...
View ArticleResearchers improve vbectors for delivering hFVIII gene therapy to treat...
A new study examined 42 combinations of promoters and enhancers for human factor VIII (hFVIII) gene expression to identify the optimal adeno-associated virus (AAV)-based gene therapy delivery vector...
View ArticleDebate continues over human immune system's role in blocking therapeutic...
The hypothesis that memory T cells formed in response to childhood infections may be to blame for the loss of expression of therapeutic genes delivered via viral vectors has been hotly debated, but...
View ArticleNew approach makes cells resistant to HIV
Scientists at The Scripps Research Institute (TSRI) have found a way to tether HIV-fighting antibodies to immune cells, creating a cell population resistant to the virus. Their experiments under lab...
View ArticleResults of glioblastoma clinical trial show safety and clinical benefit of...
Glioblastoma is the most common brain tumor in humans and also one of the most difficult cancers to treat; patients with this type of cancer only survive about one year from time of diagnosis....
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