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.

New study links anxiety and PTSD to increased myelin in brain's gray matter

A recent study links anxiety behavior in rats, as well as post traumatic stress disorder (PTSD) in military veterans, to increased myelin - a substance that expedites communication between neurons - in areas of the brain associated with emotions and memory.

6 Jan 2022

New gene therapy addresses primary cellular deficit associated with limb-girdle muscular dystrophy 2B

Children's National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this disease.

4 Jan 2022

Structural blueprint of nanoparticles developed to target neutrophils responsible for acute lung inflammation

The COVID-19 pandemic highlighted the devastating impact of acute lung inflammation (ALI), which is part of the acute respiratory distress syndrome (ARDS) that is the dominant cause of death in COVID-19.

28 Dec 2021

Researchers identify new mechanism for DNA invasion of adenoviral COVID-19 vaccines

Adenoviruses have a linchpin protein that stabilizes their DNA until it reaches the infected cell's nucleus. The protein then detaches from the viral genome, and the virus uncoats. Only then are the genes released into the nucleus, which is necessary for the production of new viruses.

22 Dec 2021

Scientists develop new immunotherapy for cancer based on transplant rejection mechanism

A team of scientists at the University of Oslo, Oslo University Hospital Radiumhospitalet and Karolinska Institutet, led by Professor Johanna Olweus, has developed a new type of immunotherapy for cancer.

18 Dec 2021

Study determines minimally effective dose of clinical candidate gene therapy vector to treat hemophilia A

Researchers determined the minimally effective dose (MED) of a clinical candidate gene therapy vector for treating hemophilia A.

16 Dec 2021

First-of-its-kind gene therapy technique reverses severe hearing loss in mice

Hearing loss has been linked to mutations in at least 100 different genes, but up to 16 percent of genetic hearing loss can be traced to just one gene, STRC, the second most common genetic cause.

15 Dec 2021

WVU researchers explore how a benign virus can be used to treat eye diseases

Viruses have a bad reputation for a good reason. But some viruses don't harm people. Some can even help them.

15 Dec 2021

Gene therapy and diabetes drug may be treatments for late-onset retinal degeneration

Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease.

9 Dec 2021

An assessment of pH-sensitive cationic lipid nanoparticles for drug delivery

A recent review looks at the molecular design of pH-sensitive cationic lipids and their applications for various tissues and cell types.

7 Dec 2021

Cochlear implant recipients develop new bone formation that leads to long-term residual hearing loss

The majority of cochlear implant recipients develop new bone formation that adversely affects long-term hearing preservation, according to a study appearing in Radiology.

7 Dec 2021

Targeting the brain should be the focus of ALS therapy, not just spinal cord

The brain is indeed a target for treating ALS (amyotrophic lateral sclerosis), Northwestern Medicine scientists have discovered.

4 Dec 2021

Nanoengineers develop a new approach to effectively deliver mRNA into cells

Nanoengineers at the University of California San Diego have developed a new and potentially more effective way to deliver messenger RNA (mRNA) into cells.

30 Nov 2021

Researchers genetically map cell types that make up the mouse iris

Johns Hopkins Medicine researchers report they have genetically mapped the cell types that make up the mouse iris — the thin disc of pigmented tissue that, in humans, gives eyes their distinct colors.

30 Nov 2021

What does the future look like for bleeding disorders?

In this interview, we speak to Assad Haffar, the Medical and Humanitarian Aid Director at the World Federation of Hemophilia (WFH).

23 Nov 2021

How to achieve fast, accurate and reliable quantitation of AAV serotypes

In this interview, News-Medical talks to Indrani Chakraborty from GatorBio about how they have achieved accurate and reliable quantitation of AAV serotypes.

From Gator Bio, Inc. 19 Nov 2021

New trial demonstrates stable coagulation factor VIII in hemophilia A patients after gene therapy

A novel gene therapy for hemophilia A led to sustained expression of the clotting factor those patients lack, resulting in a reduction – or in some cases complete elimination – of painful and potentially life-threatening bleeding events, according to a new study led by researchers at Children's Hospital of Philadelphia.

18 Nov 2021

£375 million Government funding to improve treatment for neurodegenerative diseases

People living with neurodegenerative diseases could live longer, healthier lives due to innovative new research, following a government commitment to invest £375 million over the next 5 years.

14 Nov 2021

Study highlights the need for caution while using CRISPR-based gene therapies

A comprehensive study - conducted by researchers at Sanford Burnham Prebys, the National Cancer Institute (NCI) and other groups - has shown that gene editing, specifically gene knockout (KO), with CRISPR -Cas9 can favor cells with mutated forms of genes linked to cancer.

11 Nov 2021

UArizona researchers get closer to developing a safe, effective non-opioid pain reliever

Researchers at the University of Arizona Health Sciences are closer to developing a safe and effective non-opioid pain reliever after a study showed that a new compound they created reduces the sensation of pain by regulating a biological channel linked to pain.

10 Nov 2021