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.

Researchers develop a simple tool for making mouse models of COVID-19

Until there are effective treatments or vaccines, the COVID-19 pandemic will remain a significant threat to public health and economies around the world. A major hurdle to developing and testing new anti-viral therapies and vaccines for COVID-19 is the lack of good, widely available animal models of the disease.

16 Jun 2020

Exercise has profound protective effect in mouse models of Friedreich's ataxia

A top exercise researcher at the University of Virginia School of Medicine is urging clinical trials of exercise in patients with Friedreich's ataxia after finding that physical activity has a "profound" protective effect in mouse models of the debilitating genetic disease.

16 Jun 2020

UCLA receives nearly $14 million NIH grant to investigate immunotherapy to destroy HIV

UCLA researchers and colleagues have received a $13.65 million grant from the National Institutes of Health to investigate and further develop an immunotherapy known as CAR T, which uses genetically modified stem cells to target and destroy HIV.

16 Jun 2020

Early genetic rescue may be a potential therapy in autism spectrum disorder

A mouse study by Craig Powell, M.D., Ph.D., and colleagues suggests that early genetic rescue may be a potential therapy in autism spectrum disorder or ASD.

16 Jun 2020

Micro-dystrophin gene therapy can improve outcomes in children with Duchenne muscular dystrophy

Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy (DMD) - and initial findings suggest that the therapy can provide functional improvement that is greater than that observed under the standard of care.

16 Jun 2020

Researchers use human stem cell models to analyze degeneration in glaucoma

More than 3 million Americans have glaucoma, a serious eye condition causing vision loss. Using human stem cell models, researchers at Indiana University School of Medicine found they could analyze deficits within cells damaged by glaucoma, with the potential to use this information to develop new strategies to slow the disease process.

11 Jun 2020

Researchers treat TMC1-related deafness with gene therapy

When Wei Hsi (Ariel) Yeh was a young undergraduate student, one of her close friends went from normal hearing to complete deafness in the span of one month.

4 Jun 2020

New technique opens the door to treating genetic forms of hearing loss

Using a new genetic engineering technique, known as base editing, researchers from Boston Children's Hospital and the Broad Institute of MIT and Harvard, have restored hearing in mice with a known recessive genetic mutation.

3 Jun 2020

New advance addresses a big challenge with using CRISPR in cancer patients

A mutation unique to certain cancer tumors is a potential homing beacon for safely deploying CRISPR gene editing enzymes to disarm DNA that makes cancer cells resistant to treatment, while ignoring the gene in normal cells where it's critical to healthy function, according to a new study from ChristianaCare's Gene Editing Institute in the journal Molecular Cancer Research.

2 Jun 2020

CSL Behring and Seattle Children's partner to develop gene therapies for primary immunodeficiency diseases

Seattle Children's Research Institute, one of the top pediatric research institutions in the world, and global biotechnology leader CSL Behring announced a strategic alliance to develop stem cell gene therapies for primary immunodeficiency diseases.

2 Jun 2020

Microperimetry can help determine the efficacy of novel treatments for macular diseases

Stargardt disease is an inherited eye disease. Its onset is typically in adolescence and it is among the leading causes of blindness in this age group. Symptoms range from severe loss of sharp vision to almost complete loss of sight.

2 Jun 2020

Toshiba and Shinshu University jointly develop a 'tumor-tropic liposome technology' for gene therapy

Toshiba Corporation and a team led by Professor Yozo Nakazawa at the Department of Pediatrics, Shinshu University, have together developed a “tumor-tropic liposome technology” for gene therapy. The technology uses unique, nano-sized biodegradable liposomes developed by Toshiba to accurately and efficiently deliver therapeutic genes to targeted cancer cells, and achieves safer gene delivery than viruses used as carriers.

30 May 2020

Using gene therapy to help the body to make its own cancer drugs

Imagine that we could instruct our bodies to make the drugs they need themselves. The gene technology that makes this possible is called mRNA therapy, which may become a major tool in the treatment of multiple diseases.

29 May 2020

Nanoscope received NIH grant to advance ultrafast laser-based gene delivery to the retina

The Nanoscope team has developed Multi-Characteristic Opsins to sensitize cells toward low level of white light so that vision is improved at ambient room light.

28 May 2020

Frog brain discovery may pave the way for repairing birth defects in humans

A team of researchers from Tufts University, Universitat de Valencia, and Harvard University has discovered that the brains of developing frog embryos damaged by nicotine exposure can be repaired by using “ionoceuticals” that can help in the recovery of bioelectric patterns in the embryo.

28 May 2020

Researchers study how brain cancer cells acquire resistance to radiation

In a vertical climb to avoid collision with a towering mountain, a plane ejects cargo to gain altitude. Investigators at the University of Minnesota showed that cancer cells perform similar feats in escaping the killing effects of radiation.

14 May 2020

A 'window of vulnerability' for re-infection with HIV after stem cell transplantation

Some people with HIV need to undergo an allogeneic stem cell transplantation in order to treat different types of blood cancer. Most of the patient's immune cells are eliminated during these transplantations.

14 May 2020

Ori Biotech and CellGenix to collaborate in order to promote innovation within closed-system cell and gene therapy manufacturing

Ori Biotech Ltd announced a partnership to achieve closed-system preparation and the addition of CellGenix media and growth factors within CGT manufacturing.

14 May 2020

Gene therapy helps build muscle mass and reduces severity of osteoarthritis in mice

Exercise and physical therapy often are recommended to help people who have arthritis. Both can strengthen muscle -; a benefit that also can reduce joint pain.

8 May 2020

Tissue engineering can play an important role during COVID-19 pandemic

Tissue engineering has a unique set of tools and technologies for developing preventive strategies, diagnostics, and treatments that can play an important role during the ongoing COVID-19 pandemic.

8 May 2020

Gene Therapy News and Research

Further Reading

Researchers develop a simple tool for making mouse models of COVID-19

Exercise has profound protective effect in mouse models of Friedreich's ataxia

UCLA receives nearly $14 million NIH grant to investigate immunotherapy to destroy HIV

Early genetic rescue may be a potential therapy in autism spectrum disorder

Micro-dystrophin gene therapy can improve outcomes in children with Duchenne muscular dystrophy

Researchers use human stem cell models to analyze degeneration in glaucoma

Researchers treat TMC1-related deafness with gene therapy

New technique opens the door to treating genetic forms of hearing loss

New advance addresses a big challenge with using CRISPR in cancer patients

CSL Behring and Seattle Children's partner to develop gene therapies for primary immunodeficiency diseases

Microperimetry can help determine the efficacy of novel treatments for macular diseases

Toshiba and Shinshu University jointly develop a 'tumor-tropic liposome technology' for gene therapy

Using gene therapy to help the body to make its own cancer drugs

Nanoscope received NIH grant to advance ultrafast laser-based gene delivery to the retina

Frog brain discovery may pave the way for repairing birth defects in humans

Researchers study how brain cancer cells acquire resistance to radiation

A 'window of vulnerability' for re-infection with HIV after stem cell transplantation

Ori Biotech and CellGenix to collaborate in order to promote innovation within closed-system cell and gene therapy manufacturing

Gene therapy helps build muscle mass and reduces severity of osteoarthritis in mice

Tissue engineering can play an important role during COVID-19 pandemic

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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