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.

UAB scientists explore gene therapy for frontotemporal dementia

Gene therapy has shown promise for treating a neurodegenerative disease known as frontotemporal dementia and a related condition called neuronal ceroid lipofuscinosis, according to new findings presented in the Journal of Neuroscience by researchers from the University of Alabama at Birmingham.

1 Mar 2018

Researchers describe important step toward gene therapy for patients with Sandhoff disease

Babies with the rare, deadly genetic disorder Sandhoff disease begin to miss developmental milestones just months after birth. Lacking muscle tone, they never learn to sit up, develop heads too large to lift and eventually suffer uncontrollable seizures. There is no cure.

22 Feb 2018

Scientists decode molecular structure of healthy huntingtin protein

Mutations on a single gene, the huntingtin gene, are the cause of Huntington's disease. They lead to an incorrect form of the correspondent protein.

22 Feb 2018

Study reveals biological relevance of sex in effects of D-galactose induced aging

A research study by the Universitat Autònoma de Barcelona in collaboration with the University of La Laguna demonstrates in mice the biological relevance of sex in the effects of accelerated ageing caused by a chronic treatment of D-galactose, a sugar found abundantly in milk and to a lesser extent in fruits and vegetables.

20 Feb 2018

AbbVie and Voyager Therapeutics collaborate to develop new treatments for tauopathies

AbbVie, a global biopharmaceutical company, and Voyager Therapeutics, Inc., a clinical-stage gene therapy company, today announced that they have entered into an exclusive strategic collaboration and option agreement to develop and commercialize vectorized antibodies directed against tau for the treatment of Alzheimer's disease and other neurodegenerative diseases.

20 Feb 2018

Therapeutic target for glaucoma could have treatment ramifications for Alzheimer’s and Parkinson’s

A therapeutic target to preserve vision in glaucoma patients could have treatment ramifications for age-related neurodegenerative disorders like Alzheimer's and Parkinson's, according to findings released today in the Proceedings of the National Academy of Sciences.

20 Feb 2018

UNC gene therapy scientists uncover viral barcode to penetrate blood-brain barrier

Gene therapies promise to revolutionize the treatment of many diseases, including neurological diseases such as ALS. But the small viruses that deliver therapeutic genes can have adverse side effects at high doses.

11 Feb 2018

Human iPS cell-derived inner ear cells may improve hereditary hearing loss

A Japanese research group has successfully grafted human iPS cell-derived inner ear cells that express human-derived proteins into the inner ears of embryonic mice.

8 Feb 2018

Novel method can edit genes with substantially fewer errors

Gene therapy is an emerging strategy to treat diseases caused by genetic abnormalities. One form of gene therapy involves the direct repair of a defective gene, using genome-editing technology such as CRISPR-Cas9. Despite its therapeutic potential, genome editing can also introduce unwanted and potentially harmful genetic errors that limit its clinical feasibility.

5 Feb 2018

Researchers develop gene therapy for idiopathic pulmonary fibrosis

Researchers have successfully identified the genetic cause of idiopathic pulmonary fibrosis and have shown success in trials of a new gene therapy.

1 Feb 2018

Stealth gene shuttles open up new avenues to treat aggressive cancers

Viruses have their own genetic material and can infect human cells in a very specific manner. They will then reproduce as directed by their own genes but using the resources of the host cell.

31 Jan 2018

Researchers use gene therapy that lengthens telomeres to cure pulmonary fibrosis in mice

Idiopathic pulmonary fibrosis is a potentially lethal disease associated with the presence of critically short telomeres, currently lacking effective treatment.

30 Jan 2018

Researchers describe severe toxicity with AAV9-based high-dose gene therapy in animal models

Researchers have described severe, life-threatening toxicity in monkeys and piglets given high doses of gene therapy delivered using an adeno-associated virus (AAV9) vector capable of accessing spinal cord neurons.

30 Jan 2018

Chinese researchers develop multifunctional vehicle to transport gene therapeutics

Gene editing is one of the hottest topics in cancer research. A Chinese research team has now developed a gold-nanoparticle-based multifunctional vehicle to transport the "gene scissors" to the tumor cell genome.

22 Jan 2018

Research shows how Zika virus damages placenta to cause malformations in babies

Though the Zika virus is widely known for a recent outbreak that caused children to be born with microencephaly, or having a small head, and other malformations, scientists have struggled to explain how the virus affects a baby as it grows in the womb.

19 Jan 2018

New vaccine approach offers effective protection against tuberculosis

A unique platform that resulted in a promising HIV vaccine has also led to a new, highly effective vaccine against tuberculosis that is moving toward testing in humans.

18 Jan 2018

Researchers discover why exercise is good and high blood pressure is bad for the heart

When the heart is put under stress during exercise, it is considered healthy. Yet stress due to high blood pressure is bad for the heart. Why? And is this always the case? Researchers of the German Centre for Cardiovascular Research and of Heidelberg University Hospital have obtained new findings which indicate that a previously undetected signal pathway causes or protects from heart failure - depending on the type of stress.

9 Jan 2018

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

Sangamo Therapeutics, Inc. and Pfizer Inc. today announced a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.

8 Jan 2018

UC Davis expert expresses caution on genetically engineered HSCs

In a commentary published in the Jan. 4 issue of the New England Journal of Medicine, UC Davis researcher William Murphy expressed cautious optimism about efforts to genetically engineer hematopoietic stem cells (HSCs) to temporarily resist cell death during transplantation.

4 Jan 2018

Genetic labeling helps researchers track behavior of blood cells

By tagging bone marrow cells of mice with a genetic label, or barcode, researchers were able to track and describe the family tree of individual blood cells as they form in their natural environment.

4 Jan 2018

Gene Therapy News and Research

Further Reading

UAB scientists explore gene therapy for frontotemporal dementia

Researchers describe important step toward gene therapy for patients with Sandhoff disease

Scientists decode molecular structure of healthy huntingtin protein

Study reveals biological relevance of sex in effects of D-galactose induced aging

AbbVie and Voyager Therapeutics collaborate to develop new treatments for tauopathies

Therapeutic target for glaucoma could have treatment ramifications for Alzheimer’s and Parkinson’s

UNC gene therapy scientists uncover viral barcode to penetrate blood-brain barrier

Human iPS cell-derived inner ear cells may improve hereditary hearing loss

Novel method can edit genes with substantially fewer errors

Researchers develop gene therapy for idiopathic pulmonary fibrosis

Stealth gene shuttles open up new avenues to treat aggressive cancers

Researchers use gene therapy that lengthens telomeres to cure pulmonary fibrosis in mice

Researchers describe severe toxicity with AAV9-based high-dose gene therapy in animal models

Chinese researchers develop multifunctional vehicle to transport gene therapeutics

Research shows how Zika virus damages placenta to cause malformations in babies

New vaccine approach offers effective protection against tuberculosis

Researchers discover why exercise is good and high blood pressure is bad for the heart

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

UC Davis expert expresses caution on genetically engineered HSCs

Genetic labeling helps researchers track behavior of blood cells

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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