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 class of antisense oligonucleotide therapeutics tested in humans

A first-in-human study with a new class of antisense oligonucleotide therapeutics showed the ability to target the RNA-silencing drug to the liver, resulting in improved potency and safety at therapeutic doses. The design and results of this trial, conducted in healthy human volunteers are reported in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

28 Jan 2019

CMV vaccine provides a new way to fight malaria

Early research on a new approach to protecting against malaria is offering promising, potentially long-lasting results against the persistent parasite that sickens hundreds of millions people each year.

24 Jan 2019

Mouse studies challenge 'inhibition' theory of autism

A detailed study of four mouse models of autism challenges the most common assumption about what goes wrong in brain circuits to cause disease symptoms.

21 Jan 2019

Scientists develop new gene therapy that prevents axon destruction in mice

Nerve axons serve as the wiring of the nervous system, sending electrical signals that control movement and sense of touch.

18 Jan 2019

New method modifies blood stem cells to reverse genetic mutation that causes IPEX

UCLA researchers led by Dr. Donald Kohn have created a method for modifying blood stem cells to reverse the genetic mutation that causes a life-threatening autoimmune syndrome called IPEX.

11 Jan 2019

Research provides clues for understanding and optimizing bone marrow transplants

Bone marrow transplants, which involve transplanting healthy blood stem cells, offer the best treatment for many types of cancers, blood disorders and immune diseases. Even though 22,000 of these procedures are performed each year in the US, much remains to be understood about how they work.

9 Jan 2019

GE Healthcare and VUMC announce partnership to enable safer, more precise cancer immunotherapies

GE Healthcare and Vanderbilt University Medical Center today announced a five-year partnership to enable safer and more precise cancer immunotherapies. Multiple diagnostic tools will be developed to help predict both the efficacy of an immunotherapy treatment and its adverse effects for a specific patient before the therapy is administered.

7 Jan 2019

New study describes various gene therapy strategies for treating Parkinson's disease

Parkinson's disease is a neurodegenerative disorder characterized by motor and nonmotor deficits majorly caused by the loss of dopaminergic cells in the Substantia Nigra pars compacta as well as the destruction of nigrostriatal pathway.

28 Dec 2018

Researchers identify hundreds of new genes linked to blindness in lab mice

Hundreds of new genes linked to blindness and other vision disorders have been identified in a screen of mouse strains. Many of these genes are likely important in human vision and the results could help identify new causes of hereditary blindness in patients.

24 Dec 2018

New project develops more efficient treatment approaches for cardiovascular diseases

Within the framework of a European collaborative research project, Oliver Müller, professor at the Faculty of Medicine at Kiel University and Consultant at the Department of Internal Medicine III, University Medical Center Schleswig-Holstein, Campus Kiel, will receive 1.4 million Euros for the project "CardioReGenix: Development of Next-Generation Gene Therapies for Cardiovascular Disease".

21 Dec 2018

Experimental gene therapy extends lifespan of mice suffering from untreatable mitochondrial disease

Researchers at the University of Helsinki and Folkhälsan Research Center have, together with their collaborators, for the first time demonstrated in a mouse model that partial restoration of respiratory chain function in mitochondria, which serve as cellular power plants, may be sufficient to entirely prevent symptoms of a severe mitochondrial disorder.

20 Dec 2018

UI study leads to better understanding of signaling capacity between neurons

The fundamental process of information transfer from neuron to neuron occurs through a relay of electrical and chemical signaling at the synapse, the junction between neurons. Electrical signals, called action potentials, cause voltage-gated calcium channels on the presynaptic neuron to open.

18 Dec 2018

New discovery will improve the safety and predictability of CRISPR

Scientists have discovered a simple set of rules that can be used to precisely predict how DNA will be modified by the CRISPR gene editing technique.

13 Dec 2018

Study provides new understanding of mysterious 'hereditary swelling'

An upper arm that swells to twice its normal size. A stomach that inexplicably swells up causing colic pain, vomiting or diarrhea. Or a life-threatening variant with severe swelling of the face, lips, tongue and larynx, bringing the risk of obstructed airways and death by suffocation.

11 Dec 2018

Lund University researchers succeed in obtaining dendritic cells by direct reprogramming

For the first time, a research team at Lund University in Sweden has successfully reprogrammed mouse and human skin cells into immune cells called dendritic cells.

9 Dec 2018

Elevated hormone flags liver disease in mice with serious genomic disorder

Researchers have discovered that a hormone, fibroblast growth factor 21 (FGF21), is extremely elevated in mice with liver disease that mimics the same condition in patients with methylmalonic acidemia (MMA), a serious genomic disorder.

6 Dec 2018

CHOP celebrates EC approval of first and only gene therapy for patients with inherited retinal disease

Children's Hospital of Philadelphia celebrates a pivotal moment in medicine: approval by the European Commission of LUXTURNA (voretigene neparvovec), the first and only gene therapy for patients with an inherited retinal disease, last month.

6 Dec 2018

Physician-scientists present updated efficacy data on CAR T-cell gene immunotherapy

Physician-scientists from Children's Hospital of Philadelphia presented updated efficacy and safety data on Kymriah--the first-ever U.S. Food and Drug Administration approved personalized CAR T-cell gene immunotherapy for aggressive blood cancers at the 60th American Society of Hematology annual meeting, as well as first-of-its-kind research on overcoming CAR T-cell resistance.

5 Dec 2018

Advances in gene therapy for inherited blood disorders

Gene therapy holds a lot of promise in medicine. If we could safely alter our own DNA, we might eliminate diseases our ancestors passed down to us.

4 Dec 2018

NanoBioCel group develops lipid vesicles that can genetic material efficiently and viably

In a piece of research conducted in collaboration, the NanoBioCel group of the UPV/EHU's Faculty of Pharmacy, and the University of Elche have designed some niosomes, which are lipid vesicles for use in gene therapies designed to treat diseases of the central nervous system.