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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.
Innovative approach to treating AAT deficiency

Innovative approach to treating AAT deficiency

Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin (AAT)--which is missing or nonfunctional in the genetic disorder AAT deficiency--into cells in the laboratory, enabling the cells to produce highly functional AAT. [More]
Infants born with mutation in PLVAP gene develop severe protein losing enteropathy

Infants born with mutation in PLVAP gene develop severe protein losing enteropathy

Newborn children born with a mutation in the Plasmalemma Vesicle Associated Protein (PLVAP) gene develop severe protein losing enteropathy, according to a case study1 published in Cellular and Molecular Gastroenterology and Hepatology, the basic science journal of the American Gastroenterological Association. [More]
New TCR therapy demonstrates encouraging clinical responses in patients with multiple myeloma

New TCR therapy demonstrates encouraging clinical responses in patients with multiple myeloma

Results from a clinical trial investigating a new T cell receptor (TCR) therapy that uses a person's own immune system to recognize and destroy cancer cells demonstrated a clinical response in 80 percent of multiple myeloma patients with advanced disease after undergoing autologous stem cell transplants (ASCT). [More]
Adaptimmune’s NY-ESO-1 TCR-engineered T-cell therapy mediates sustained antitumor effects in multiple myeloma patients

Adaptimmune’s NY-ESO-1 TCR-engineered T-cell therapy mediates sustained antitumor effects in multiple myeloma patients

Adaptimmune Therapeutics plc, a clinical stage biopharmaceutical company focused on the use of T-cell therapy to treat cancer, today announced that data from its Phase I/II study of its affinity enhanced T-cell receptor therapeutic targeting the NY-ESO-1 cancer antigen in patients with multiple myeloma has been published in Nature Medicine. [More]
Rowan University, Bamboo Therapeutics partner to combat Canavan disease

Rowan University, Bamboo Therapeutics partner to combat Canavan disease

Rowan University and Bamboo Therapeutics, Inc. have entered into an asset transfer agreement to commercialize a novel gene therapy for the treatment of Canavan disease. A rare but devastating neurological disease that tragically takes a child's life by age 10, Canavan disease is one of the most common and complex degenerative cerebral diseases in infants. [More]
Gene therapy used for sight restoration also strengthens visual pathways in the brain

Gene therapy used for sight restoration also strengthens visual pathways in the brain

Since 2007, clinical trials using gene therapy have resulted in often-dramatic sight restoration for dozens of children and adults who were otherwise doomed to blindness. Now, researchers from the Perelman School of Medicine at the University of Pennsylvania and The Children's Hospital of Philadelphia, have found evidence that this sight restoration leads to strengthening of visual pathways in the brain, published this week in Science Translational Medicine. [More]
Research breakthrough opens door to a world of regenerative medicine for treating mitochondrial disease

Research breakthrough opens door to a world of regenerative medicine for treating mitochondrial disease

A study led by Shoukhrat Mitalipov, Ph.D., and Hong Ma, M.D., Ph.D., at the Center for Embryonic Cell and Gene Therapy at Oregon Health & Science University and the Oregon National Primate Research Center has revealed the first critical step in developing novel gene and stem cell therapy treatments for patients with mitochondrial disease. [More]
Gene therapy provides life-long protection to photoreceptor cells in animal model of retinitis pigmentosa

Gene therapy provides life-long protection to photoreceptor cells in animal model of retinitis pigmentosa

A collaboration between scientists in the UK and the USA has shown that gene therapy can give life-long protection to the light-sensitive photoreceptor cells responsible for colour vision in a mouse model of the most common inherited eye disorder. [More]
New study reveals highly promising approach to coating tissue engineered constructs

New study reveals highly promising approach to coating tissue engineered constructs

A new study showing the ability to apply a thin coating of viable respiratory epithelial cells to tissue engineered constructs using a commercially available spray device is especially promising for therapeutic approaches in development to repair or replace challenging structures such as trachea or bronchi. [More]
Hiroshima University researchers reveal molecular mechanisms involved in neuropathic pain

Hiroshima University researchers reveal molecular mechanisms involved in neuropathic pain

A research group from Hiroshima University demonstrated that the downregulation of spinal astrocyte connexin43 (Cx43) expression causes sustained neuropathic pain following peripheral nerve injury. Controlling the Cx43 expression using pharmacological approaches or gene therapy might serve as novel therapeutic strategies ameliorate neurological disorders in general and neuropathic pain in particular. [More]
Novel gene therapy control system regulates expression of therapeutic transgenes

Novel gene therapy control system regulates expression of therapeutic transgenes

Korean researchers have described a novel control system to regulate the expression of a therapeutic transgene by targeting the passenger strand of a microRNA (miR-122) linked to the transgene. [More]
Researchers reveal how certain lipids help adenoviruses to enter the cell

Researchers reveal how certain lipids help adenoviruses to enter the cell

Adenoviruses cause numerous diseases, such as eye or respiratory infections, and they are widely used in gene therapy. Researchers from the University of Zurich have now discovered how these viruses penetrate the cells, a key step for infection and gene delivery. The cell unwillingly supports virus entry and infection by providing lipids that are normally used to repair damaged membranes. [More]
TSRI study reveals alternative approach to current anti-HIV strategies

TSRI study reveals alternative approach to current anti-HIV strategies

HIV-infected patients remain on antiretroviral therapy for life because the virus survives over the long-term in infected dormant cells. Interruption of current types of antiretroviral therapy results in a rebound of the virus and clinical progression to AIDS. [More]
Findings reveal new pathway to develop effective treatments and therapies for asthma, allergy

Findings reveal new pathway to develop effective treatments and therapies for asthma, allergy

Investigators have discovered the precise molecular steps that enable immune cells implicated in certain forms of asthma and allergy to develop and survive in the body. The findings from Weill Cornell Medical College reveal a new pathway that scientists could use to develop more effective treatments and therapies for the chronic lung disorder. [More]
Gene therapy improves lung function in patients with cystic fibrosis

Gene therapy improves lung function in patients with cystic fibrosis

Gene therapy was shown to provide significant benefits for patients with cystic fibrosis in a recent clinical study published in The Lancet Respiratory Medicine journal. [More]
Gene therapy for cystic fibrosis shows significant benefit in lung function

Gene therapy for cystic fibrosis shows significant benefit in lung function

For the first time gene therapy for cystic fibrosis has shown a significant benefit in lung function compared with placebo, in a phase 2 randomised trial published in The Lancet Respiratory Medicine journal. The technique replaces the defective gene response for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells. [More]
New NEI study shows that microglia can accelerate damage wrought by blinding eye disorders

New NEI study shows that microglia can accelerate damage wrought by blinding eye disorders

Spider-like cells inside the brain, spinal cord and eye hunt for invaders, capturing and then devouring them. These cells, called microglia, often play a beneficial role by helping to clear trash and protect the central nervous system against infection. But a new study by researchers at the National Eye Institute (NEI) shows that they also accelerate damage wrought by blinding eye disorders, such as retinitis pigmentosa. [More]
Canada's first human gene therapy trial for choroideremia now underway at Royal Alexandra Hospital

Canada's first human gene therapy trial for choroideremia now underway at Royal Alexandra Hospital

Canada's first human gene therapy trial for eyes -- the replacement of a faulty gene with a healthy one -- is now underway at the Royal Alexandra Hospital to preserve and potentially restore vision for people with a genetic disorder that leaves them blind by middle age. [More]
Two physician-scientists in search for better Alzheimer's disease treatment

Two physician-scientists in search for better Alzheimer's disease treatment

Two of the nation's leading physician-scientists in the search to better understand and treat Alzheimer's disease - William Mobley, MD, PhD, and Michael Rafii, MD, PhD - have been named interim co-directors of the Alzheimer's Disease Cooperative Study (ADCS), a major initiative formed in 1991 as a cooperative agreement between the National Institute on Aging (NIA) and the University of California, San Diego. [More]
Innovative gene transfer-based treatment for kids with giant axonal neuropathy

Innovative gene transfer-based treatment for kids with giant axonal neuropathy

University of North Carolina School of Medicine researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN). [More]
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