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.

Single-particle method helps determine isoelectric point of viruses

Viruses are not easy to characterize. But we need to because being able to quickly predict the surface charge of viruses opens up new possibilities for vaccine purification and making gene therapy treatments for eye diseases and muscular dystrophy.

8 Jan 2020

Researchers report first structural model for key enzyme associated with inherited eye diseases

Researchers at the University of New Hampshire have reported the first structural model for a key enzyme, and its activating protein, that can play a role in some genetically inherited eye diseases like retinitis pigmentosa and night blindness.

8 Jan 2020

Biodegradable nanoparticle can deliver 'suicide gene' to pediatric brain tumor cells in mice

Johns Hopkins researchers report that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a "suicide gene" to pediatric brain tumor cells implanted in the brains of mice.

8 Jan 2020

Dog study suggests that AAV in gene therapy could induce cancer

A gene therapy study in dogs has stirred up old fears that by using AAV in gene therapy, it may be increasing cancer chances.

8 Jan 2020

Mayo Clinic researchers seek radical new approach to treat cocaine addition

In a radical new approach to treat cocaine addition, researchers at the Mayo Clinic are seeking approval for first-in-human studies of a single-dose gene therapy.

7 Jan 2020

Using CRISPR to switch off pain gene becomes a possibility with new study

A pathbreaking study reports success in disabling an important gene that is responsible for inducing the sensation of pain.

7 Jan 2020

Study shows safety, long-term beneficial impact of gene therapy for patients with Hemophilia A

A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK.

6 Jan 2020

Study shows the feasibility of using gene therapy to treat chronic traumatic encephalopathy

A new study shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy.

3 Jan 2020

Inserting end-modified DNA fragments with CRISPR-Cas9 yields highest efficiency

University of Illinois researchers achieved the highest reported rates of inserting genes into human cells with the CRISPR-Cas9 gene-editing system, a necessary step for harnessing CRISPR for clinical gene-therapy applications.

23 Dec 2019

IU researchers identify potential target to improve functional recovery after spinal cord injury

Researchers at Indiana University School of Medicine have made several novel discoveries in the field of spinal cord injuries. Most recently, the team led by Xiao-Ming Xu, PhD, has been working to determine how to activate movement after a spinal cord injury at the ninth thoracic level, where nerve fibers from the brain down to the spinal cord are interrupted.

22 Dec 2019

Study uncovers a previously unknown subtype of muscle stem cells

Scientists have discovered a new subtype of muscle stem cells. These cells have the ability to build and regenerate new muscles, making them interesting targets for the development of gene therapies.

21 Dec 2019

Scientists explain how a common virus causes havoc on bone marrow, organ transplant patients

For the first time, scientists have discovered the molecular mechanism behind how a common virus can wreak havoc on bone marrow and organ transplant patients, according to a paper published in the journal Cell & Host Microbe.

19 Dec 2019

Worse long-term control of blood sugar increases risks in diabetic trauma patients

In trauma patients with diabetes, poorer long-term control of blood glucose levels is linked to a higher risk of death and trauma-related complications, reports a study in SHOCK: Injury, Inflammation, and Sepsis: Laboratory and Clinical Approaches, Official Journal of the Shock Society.

18 Dec 2019

Nature’s own gene therapy

A brand-new technique of gene therapy was reported on December 16, 2019, in the journal Nature Biomedical Engineering. The scientists made use of the potential of human cells to produce biological nanoparticles chockful of edited genes, so that they could insert beneficial genes into people suffering from various disease processes.

17 Dec 2019

New gene therapy has potential to reverse disease processes

Scientists have developed a new gene-therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that has the potential to reverse disease processes.

17 Dec 2019

Neuroscientists develop new method for uniquely targeted gene therapy

Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe that the new technology can be likened to dramatically accelerating evolution from millions of years to weeks.

13 Dec 2019

University Of Iowa wins $18 million grant to continue study on Huntington's disease

Peg Nopoulos, MD, chair of psychiatry and professor of psychiatry, neurology, and pediatrics in the University of Iowa Carver College of Medicine, has received a five-year, $18 million grant from the National Institute of Neurological Disorders and Stroke to continue and expand a decade-long study on brain development in children at risk for developing Huntington's Disease.

11 Dec 2019

New partnership enables launch of POCare cell therapy platform within HYGEIA Group’s hospital network

Orgenesis Inc., a leading cell and gene therapy enabling company providing centralized CDMO manufacturing and development services through its subsidiary Masthercell Global, Inc., as well as localized point-of-care development and processing centers for therapeutic treatments, today announces a strategic partnership agreement between the HYGEIA Group and the Theracell–Orgenesis joint venture.

9 Dec 2019

Scientists uncover new link between pathways involved in RNA degradation

A recent study by a team of scientists in Korea reveals new findings about how various systems involved in cellular surveillance interact. This research is the first to identify a "cross-talk" molecule between these systems. Because these pathways are involved in fighting toxic cellular or foreign substances, the study has various potential applications in antiviral development, gene therapy, and agriculture.

6 Dec 2019

US pharmaceutical industry continues to shine according to CPhI 2019 Annual Report

The US pharmaceutical industry continues to lead its global peers in terms of innovation and competitiveness, while still ranking high in overall growth potential despite its mature status, according to the results of a survey of 350 global pharmaceutical industry executives published as part of the recently released CPhI 2019 Annual Report.

From LabVantage Solutions 6 Dec 2019

Gene Therapy News and Research

Further Reading

Single-particle method helps determine isoelectric point of viruses

Researchers report first structural model for key enzyme associated with inherited eye diseases

Biodegradable nanoparticle can deliver 'suicide gene' to pediatric brain tumor cells in mice

Dog study suggests that AAV in gene therapy could induce cancer

Mayo Clinic researchers seek radical new approach to treat cocaine addition

Using CRISPR to switch off pain gene becomes a possibility with new study

Study shows safety, long-term beneficial impact of gene therapy for patients with Hemophilia A

Study shows the feasibility of using gene therapy to treat chronic traumatic encephalopathy

Inserting end-modified DNA fragments with CRISPR-Cas9 yields highest efficiency

IU researchers identify potential target to improve functional recovery after spinal cord injury

Study uncovers a previously unknown subtype of muscle stem cells

Scientists explain how a common virus causes havoc on bone marrow, organ transplant patients

Worse long-term control of blood sugar increases risks in diabetic trauma patients

Nature’s own gene therapy

New gene therapy has potential to reverse disease processes

Neuroscientists develop new method for uniquely targeted gene therapy

University Of Iowa wins $18 million grant to continue study on Huntington's disease

New partnership enables launch of POCare cell therapy platform within HYGEIA Group’s hospital network

Scientists uncover new link between pathways involved in RNA degradation

US pharmaceutical industry continues to shine according to CPhI 2019 Annual Report

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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