Gene Therapy News and Research

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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.

Study suggests potential means of stalling or even reversing diabetes-related blindness

Johns Hopkins researchers have discovered a cell signaling pathway in mice that triggers vision loss in patients with diabetic retinopathy and retinal vein occlusion – diseases characterized by the closure of blood vessels in the retina, leading to blindness.

3 Jan 2018

Gene therapy with CAR T-cells could create long-term immunity from HIV

Through gene therapy, researchers engineered blood-forming stem cells (hematopoietic stem/progenitor cells, or HSPCs) to carry chimeric antigen receptor (CAR) genes to make cells that can detect and destroy HIV-infected cells.

29 Dec 2017

FDA approves new gene therapy targeting rare form of inherited vision loss

The U.S. Food and Drug Administration today approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness.

19 Dec 2017

A guide to Severe Combined Immunodeficiency Disease, review released

A new review published today in the Canadian Medical Association Journal provides guidance on Severe Combined Immunodeficiency Disease (SCID), a deadly and rare disease that is potentially curable if detected early.

18 Dec 2017

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

A new review provides guidance on a deadly, but rare, disease that is potentially curable if identified early. Severe combined immunodeficiency disease (SCID), known as the "bubble boy disease" in the 1970s, is treatable with a stem cell transplant, gene therapy and other treatments if identified at birth or soon after.

18 Dec 2017

Gene that could be associated with reduced pain response found

Researchers found an Italian family, the Marsilis that have six individuals who respond significantly differently to pain than others. This includes a 78-year-old lady and her two daughters and their three children.

18 Dec 2017

Rare gene mutation gives rise to low sensitivity to pain

Researchers at University College London have identified a rare gene mutation in an Italian family who have little sensitivity to some forms of pain. Chronic pain caused by conditions such as lower back pain and osteoarthritis is one of the most prominent causes of disability worldwide, as assessed by the Global Burden of Disease reviews.

15 Dec 2017

Groundbreaking gene therapy trial brings cure for hemophilia closer

A 'cure' for hemophilia is one step closer, following results of a groundbreaking gene therapy trial led by the NHS in London.

14 Dec 2017

New, more easily administered therapies offer benefits for bleeding and clotting disorders

In three studies being presented today during the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta, researchers report remarkable benefits from new, more easily administered therapies for bleeding and clotting disorders.

11 Dec 2017

St. Jude gene therapy offers hope for infants with 'bubble boy' disease

Early evidence suggests that gene therapy developed at St. Jude Children's Research Hospital will lead to broad protection for infants with the devastating immune disorder X-linked severe combined immunodeficiency disorder.

11 Dec 2017

Gene therapy shows promising effect against blood clots, study states

A new study conducted in the Children's Hospital Of Philadelphia (CHOP) reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B.

8 Dec 2017

UA researcher awarded $2.3 million NIH grant to develop new treatments for depression

A University of Arizona College of Medicine-Phoenix researcher has received a $2.3 million grant from the National Institutes of Health to develop new treatments for depression.

7 Dec 2017

Overcoming Challenges of Assays of Complex Cellular Models with Real-Time Live-Cell Analysis

There's a continued drive towards making in vitro assays ever more translational towards in vivo models and ultimately the clinic. This ties in with the resurgence of phenotypic screening and is a response to the perceived poor translation of the traditional simple cell-based assays, often developed to study just single protein targets.

From Sartorius 5 Dec 2017

Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

Orchard Therapeutics, a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies, today announced the opening of a second facility for technical operations in the San Francisco Bay Area.

1 Dec 2017

Study focuses on improving pharmacokinetic properties of therapeutic aptamers

Designing new therapeutic DNA aptamers with diverse side chains can improve their ability to interact with targets, and a new study describes characteristics of these side chains that may determine how long the aptamers remain in the bloodstream.

30 Nov 2017

Powerful digital model replicates biological activity that precedes sudden cardiac death

Some heart disease patients face a higher risk of sudden cardiac death, which can happen when an arrhythmia -- an irregular heartbeat-- disrupts the normal electrical activity in the heart and causes the organ to stop pumping.

29 Nov 2017

Virus Painting, Lipids and Single Particle Charge

Various viral infections still pose a great threat to human and animal health. A wide range of viral species are surrounded by a lipid shell, the envelope, containing viral and cellular proteins.

From Izon Science 27 Nov 2017

Researchers convert natural bacterial immune system into microscopic data recorder

Through a few clever molecular hacks, researchers at Columbia University Medical Center have converted a natural bacterial immune system into a microscopic data recorder, laying the groundwork for a new class of technologies that use bacterial cells for everything from disease diagnosis to environmental monitoring.

23 Nov 2017

Study identifies signaling pathway essential for growth of new blood vessels

A new study led by researchers at Sanford Burnham Prebys Medical Discovery Institute (SBP) identifies a signaling pathway that is essential for angiogenesis, the growth of new blood vessels from pre-existing vessels.

23 Nov 2017

Amish people live longer due to mutation in blood clotting gene

Scientists have stumbled upon a genetic mutation that is found among the Indiana Amish community people that leads to impaired or defective blood clotting but predicts a longer life span for them. The study was published this week in Science Advances.

19 Nov 2017

Gene Therapy News and Research

Further Reading

Study suggests potential means of stalling or even reversing diabetes-related blindness

Gene therapy with CAR T-cells could create long-term immunity from HIV

FDA approves new gene therapy targeting rare form of inherited vision loss

A guide to Severe Combined Immunodeficiency Disease, review released

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

Gene that could be associated with reduced pain response found

Rare gene mutation gives rise to low sensitivity to pain

Groundbreaking gene therapy trial brings cure for hemophilia closer

New, more easily administered therapies offer benefits for bleeding and clotting disorders

St. Jude gene therapy offers hope for infants with 'bubble boy' disease

Gene therapy shows promising effect against blood clots, study states

UA researcher awarded $2.3 million NIH grant to develop new treatments for depression

Overcoming Challenges of Assays of Complex Cellular Models with Real-Time Live-Cell Analysis

Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

Study focuses on improving pharmacokinetic properties of therapeutic aptamers

Powerful digital model replicates biological activity that precedes sudden cardiac death

Virus Painting, Lipids and Single Particle Charge

Researchers convert natural bacterial immune system into microscopic data recorder

Study identifies signaling pathway essential for growth of new blood vessels

Amish people live longer due to mutation in blood clotting gene

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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