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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
Novel gene therapy can treat pulmonary hypertension linked with heart failure

Novel gene therapy can treat pulmonary hypertension linked with heart failure

Scientists have used a novel gene therapy to halt the progression of pulmonary hypertension, a form of high blood pressure in the lung blood vessels that is linked to heart failure, according to a study led by Roger J. Hajjar, MD, Professor of Medicine and Director of the Cardiovascular Research Center at the Icahn School of Medicine at Mount Sinai. [More]
Pioneering gene therapy may lead to potential cure for common causes of vision loss

Pioneering gene therapy may lead to potential cure for common causes of vision loss

Pioneering gene therapy has restored some vision to patients with a rare form of genetic blindness for as long as four years, raising hopes it could be used to cure common causes of vision loss, new University of Oxford research published today shows. [More]
New study on retroviral DNA could help improve treatments for HIV infection

New study on retroviral DNA could help improve treatments for HIV infection

When retroviruses such as HIV infect a cell, they first make a copy of their RNA genome in the form of DNA. The relatively short viral DNA strand then moves to the cell nucleus, where it inserts itself into the host cell's DNA. [More]
Modernization of screening methods in sperm banks could protect future children from highly heritable diseases

Modernization of screening methods in sperm banks could protect future children from highly heritable diseases

U.S. sperm banks perform genetic testing to screen for and disqualify carriers of a limited number of recessive disease mutations, but more comprehensive and affordable DNA-based screening methods are now available that can detect many more disease-causing genetic variations. [More]
Review focuses on autologous cell therapy that can be applied to cardiac surgery

Review focuses on autologous cell therapy that can be applied to cardiac surgery

The combination of cell and gene therapy is rapidly raising clinical interest, although their combination has been under investigation for several decades. This is mainly due to the availability and feasibility of clinically relevant gene delivery options. This review focuses on cell therapies for heart failure and their use combined with cardiac surgery as well as with gene therapy. [More]
Combination of nanoscale topography and triculture technology benefits large or slow-healing wounds

Combination of nanoscale topography and triculture technology benefits large or slow-healing wounds

Large or slow-healing wounds that do not receive adequate blood flow could benefit from a novel approach that combines a nanoscale graft onto which three different cell types are layered. Proper cell alignment on the nanograft allows for the formation of new blood vessel-like structures, as reported in of Tissue Engineering, Part A, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free for download on the Tissue Engineering website until May 26, 2016. [More]
First breakthrough in gene therapy against aging

First breakthrough in gene therapy against aging

In September 2015, then 44 year-old CEO of BioViva USA Inc. Elizabeth Parrish received two of her own company's experimental gene therapies: one to protect against loss of muscle mass with age, another to battle stem cell depletion responsible for diverse age-related diseases and infirmities. [More]
Novel gene therapy can improve symptoms of Bubble Boy disease in young adults

Novel gene therapy can improve symptoms of Bubble Boy disease in young adults

Adolescents and young adults with a severe inherited immunodeficiency disorder improved following treatment with novel gene therapy developed at St. Jude Children's Research Hospital and at the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. The results of this study appear today in the journal Science Translational Medicine. [More]
Study shows children with rare eye disease have greatest benefit from gene therapy

Study shows children with rare eye disease have greatest benefit from gene therapy

Scientists at Oregon Health & Science University's Casey Eye Institute and Baylor College of Medicine's Cullen Eye Institute published findings from a two-year Phase I clinical trial in the journal Ophthalmology, which showed that children had the greatest benefit from gene therapy for treatment of Leber congenital amaurosis (LCA) or severe early childhood onset retinal degeneration (SECORD). [More]
University’s blindness therapy comes a step closer following landmark licensing deal with US firm

University’s blindness therapy comes a step closer following landmark licensing deal with US firm

Hundreds of thousands of people worldwide, who have a disease that can lead to blindness, could have their sight restored after The University of Manchester entered into a technology license with Seattle-based company Acucela Inc. [More]
University's blindness therapy comes a step closer following landmark licensing deal with US firm

University's blindness therapy comes a step closer following landmark licensing deal with US firm

Hundreds of thousands of people worldwide, who have a disease that can lead to blindness, could have their sight restored after The University of Manchester entered into a technology license with Seattle-based company Acucela Inc. [More]
New study shows link between FZD7 protein and breast cancer development

New study shows link between FZD7 protein and breast cancer development

A new study shows that Frizzled7 (FZD7), a protein present on human breast epithelial cells and a component of the Wnt signaling pathway is uniquely controlled by the Notch signaling pathway, both of which play key roles in mammary gland formation and breast cancer development. [More]
Liu Laboratory at Augusta University studies exosomes using Particle Metrix’s ZetaView

Liu Laboratory at Augusta University studies exosomes using Particle Metrix’s ZetaView

Particle Metrix, developers of versatile particle characterization solutions for the life sciences, report on the work in the Liu Laboratory at Augusta University which is studying exosomes where size and concentration are critical parameters. [More]
UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

The Hemophilia and Thrombosis Treatment Center at UC San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B. [More]
Delivering microRNAs in cancer treatment: an interview with Dr Conde and Prof Artzi

Delivering microRNAs in cancer treatment: an interview with Dr Conde and Prof Artzi

microRNAs (miRs) are small endogenous noncoding RNA molecules (20–23 nucleotides) derived from imperfectly paired hairpin RNA structures naturally encoded in the genome that act specifically as triggering molecules to control translational repression or mRNA degradation. [More]
ACGT awards new research grants to take researchers to next level in cancer treatment

ACGT awards new research grants to take researchers to next level in cancer treatment

Alliance for Cancer Gene Therapy, the nation's only nonprofit dedicated exclusively to cell and gene therapies for cancer, is delighted to announce the recipients of its 2016 ACGT Young Investigator Grants. [More]
U-M researchers develop novel mouse model of children's brain cancer

U-M researchers develop novel mouse model of children's brain cancer

Children with brain cancer may soon get some help from mice with the same disease, thanks to new research from University of Michigan Medical School scientists and their colleagues. [More]

Preceyes, Nightstar partner to develop drug delivery technology for ocular surgery

Preceyes B.V. (NL) and Nightstar (UK) have entered into a collaboration for the development of a high-precision drug delivery technology in the eye. Nightstar will use the Preceyes robotic device to further refine the delivery of gene therapy to the subretinal space for a range of inherited retinal diseases. [More]
Baxalta seeks FDA approval for ADYNOVATE to treat children with hemophilia A and for use in surgical settings

Baxalta seeks FDA approval for ADYNOVATE to treat children with hemophilia A and for use in surgical settings

Baxalta Incorporated, a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, announced today that it has submitted supplemental Biologics License Applications (sBLAs) to the U.S. Food and Drug Administration seeking approval for the use of ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] to treat children under the age of 12 with hemophilia A and for use in surgical settings. [More]
New anti-ageing therapy may be effective against aplastic anaemia

New anti-ageing therapy may be effective against aplastic anaemia

Aplastic anaemia is a rare, and potentially fatal, disease of the blood, by which the bone marrow is unable to generate blood cells at the appropriate pace. Many forms of aplastic anaemia share an important link with the ageing process: the shortening of telomeres, structures that protect the ends of chromosomes. [More]
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