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 discover new, rare type of cell in the human airway

In separate studies published online in Nature on Aug. 1, two independent research teams report the discovery of a new, rare type of cell in the human airway.

2 Aug 2018

Tests show Drexel's polymer crystal nanocapsules can last longer in bloodstream

Selecting the right packaging to get precious cargo from point A to point B can be a daunting task at the post office. For some time, scientists have wrestled with a similar set of questions when packaging medicine for delivery in the bloodstream:

1 Aug 2018

Researchers use guide RNAs to improve base editing system

Researchers have used guide RNAs to improve the adenine base editing (ABE) system, used by scientists to alter genetic codes in animal models of disease.

1 Aug 2018

Rt Hon Baroness Boothroyd launches Fight for Sight campaign to raise awareness of eye health

The Rt Hon Baroness Boothroyd is launching a Fight for Sight campaign to raise awareness of eye health and the need for vital eye research.

1 Aug 2018

OHSU researchers discover disease in monkeys that mimics childhood neurodegenerative disorder

OHSU scientists have discovered a naturally occurring disease in monkeys that mimics a deadly childhood neurodegenerative disorder in people - a finding that holds promise for developing new gene therapies to treat Batten disease.

27 Jul 2018

Newer version of ALS drug may effectively treat inherited form of the disease

NIH-funded researchers delayed signs of amyotrophic lateral sclerosis in rodents by injecting them with a second-generation drug designed to silence the gene, superoxide dismutase 1.

27 Jul 2018

Gene therapy may hold potential to treat people with spinal cord injuries

Delivering a single injection of a scar-busting gene to the spinal cord of rats following injury promotes the survival of nerve cells and improves hind limb function within weeks, according to a study published April 2 in The Journal of Neuroscience.

20 Jul 2018

Staggering prices slow insurers’ coverage of CAR-T cancer therapy

Patients whose blood cancers have failed to respond to repeated rounds of chemotherapy may be candidates for a new type of gene therapy that could send their cancers into remission for years.

17 Jul 2018

Study highlights potential of fetal gene therapy to prevent lethal neurodegenerative disease

A fatal neurodegenerative condition known as Gaucher disease can be prevented in mice following fetal gene therapy, finds a new study led by UCL, the KK Women's and Children's Hospital and National University Health System in Singapore.

16 Jul 2018

CRISPR/Cas9 gene editing can cause greater genetic damage than previously thought

Scientists at the Wellcome Sanger Institute have discovered that CRISPR/Cas9 gene editing can cause greater genetic damage in cells than was previously thought. These results create safety implications for gene therapies using CRISPR/Cas9 in the future as the unexpected damage could lead to dangerous changes in some cells.

16 Jul 2018

Researchers use genome editing to reduce cholesterol levels in animal model

Using genome editing to inactivate a protein called PCSK9 effectively reduces cholesterol levels in rhesus macaques, a species of monkey, according to researchers from the Perelman School of Medicine at the University of Pennsylvania.

10 Jul 2018

Researchers use gene therapy to cure obesity and type 2 diabetes in mice

A research team from the UAB led by Professor Fatima Bosch has managed to cure obesity and type 2 diabetes in mice using gene therapy.

9 Jul 2018

Gene therapy study shows promise for chronic kidney disease

A study has shown that a viral vector can deliver genetic material to damaged kidney cells, potentially providing a treatment that can slow or reverse chronic kidney disease.

6 Jul 2018

SATB1 protein important for maintaining pluripotency of hematopoietic stem cells

Osaka University scientists demonstrate that SATB1 is important for maintaining pluripotency of hematopoietic stem cells

4 Jul 2018

City of Hope receives $3.7 million from CIRM to develop phase 1 glioblastoma trial

The California Institute for Regenerative Medicine board has awarded City of Hope $3.7 million to develop a phase 1 clinical trial for glioblastoma patients that will genetically engineer their stem cells to better tolerate chemotherapy's side effects, allowing them to receive higher doses of the therapy.

30 Jun 2018

Glucose-based dietary supplement increases muscle-force in Duchenne muscular dystrophy mouse

A dietary supplement derived from glucose increases muscle-force production in the Duchenne muscular dystrophy mouse model by 50% in ten days, according to a study conducted by researchers from Université Laval's Faculty of Medicine and Centre hospitalier universitaire de Québec Research Centre-Université Laval.

30 Jun 2018

New conference to establish roadmap for treatment-focused research on smell and taste disorders

The Monell Center, in partnership with the University of Florida Center for Smell and Taste and the Rocky Mountain Taste and Smell Center at the University of Colorado School of Medicine, will host an interdisciplinary two-day conference in November 2018 to establish a roadmap for treatment-focused research on smell and taste disorders.

27 Jun 2018

New molecular imaging method could help effectively tackle brain conditions

A new molecular imaging method can monitor the success of gene therapy in all areas of the brain, potentially allowing physicians to more effectively tackle brain conditions such as Parkinson's disease, Alzheimer's disease and multiple sclerosis.

26 Jun 2018

CellGenix completes first phase of facility expansion to meet rising demands for GMP cell culture reagents

CellGenix, a global manufacturing leader of GMP-grade raw materials for cell therapy, gene therapy and tissue-engineered products, successfully completed the first phase of its headquarters’ expansion as more customers approach late-stage clinical development and commercialization.

22 Jun 2018

Researchers show feasibility of stem cell-derived organoids for developing gene therapy vectors

A new study that compared six of the most promising adeno-associated viral gene therapy vectors in human retinal organoid models showed clear distinctions in the efficiency of gene transfer to both retinal pigment epithelial and photoreceptor cells.

14 Jun 2018

Gene Therapy News and Research

Further Reading

Researchers discover new, rare type of cell in the human airway

Tests show Drexel's polymer crystal nanocapsules can last longer in bloodstream

Researchers use guide RNAs to improve base editing system

Rt Hon Baroness Boothroyd launches Fight for Sight campaign to raise awareness of eye health

OHSU researchers discover disease in monkeys that mimics childhood neurodegenerative disorder

Newer version of ALS drug may effectively treat inherited form of the disease

Gene therapy may hold potential to treat people with spinal cord injuries

Staggering prices slow insurers’ coverage of CAR-T cancer therapy

Study highlights potential of fetal gene therapy to prevent lethal neurodegenerative disease

CRISPR/Cas9 gene editing can cause greater genetic damage than previously thought

Researchers use genome editing to reduce cholesterol levels in animal model

Researchers use gene therapy to cure obesity and type 2 diabetes in mice

Gene therapy study shows promise for chronic kidney disease

SATB1 protein important for maintaining pluripotency of hematopoietic stem cells

City of Hope receives $3.7 million from CIRM to develop phase 1 glioblastoma trial

Glucose-based dietary supplement increases muscle-force in Duchenne muscular dystrophy mouse

New conference to establish roadmap for treatment-focused research on smell and taste disorders

New molecular imaging method could help effectively tackle brain conditions

CellGenix completes first phase of facility expansion to meet rising demands for GMP cell culture reagents

Researchers show feasibility of stem cell-derived organoids for developing gene therapy vectors

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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