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.

$4 million program launched to find transformative therapies for ADNP syndrome

Researchers at the UC Davis MIND Institute are launching a $4 million program to find transformative therapies for ADNP syndrome. The rare genetic condition causes developmental delays and can affect the brain, heart, gastrointestinal system and more.

29 Mar 2022

UI neuroscientists identify fundamental biochemical mechanism underlying memory storage

A University of Iowa neuroscience research team has identified a fundamental biochemical mechanism underlying memory storage and has linked this mechanism to cognitive deficits in mouse models of Alzheimer's Disease and Related Dementias.

23 Mar 2022

A single gene can control brain cell growth in humans, research shows

Controlling how cells grow is fundamental to ensuring proper brain development and stopping aggressive brain tumors.

19 Mar 2022

Sneaking large drug-carrying biological molecules into cells using a novel drug delivery system

Scientists from Nanyang Technological University, Singapore have developed a novel method of delivering drugs into human cells using large biological molecules, by first encasing them in a protein-based microdroplet.

16 Mar 2022

Synthetic viral vector and surgical approach enable efficient gene transfer into the inner ear of primates

A synthetic viral vector and a novel surgical approach developed at Mass Eye and Ear has enabled efficient gene transfer into the inner ear of nonhuman primates, a research team working towards the development of effective treatments for hearing loss and vestibular disorders has reported.

15 Mar 2022

Bioactive small molecules enhance CRISPR/Cas9 loss-of-function editing in human cells

Researchers demonstrate enhanced loss-of-function editing of CRISPR/Cas9 in human cells.

14 Mar 2022

Researchers secure $12 million grant to develop improved therapies for hemophilia

An Indiana University School of Medicine researcher will lead a multi-institute effort toward improved therapies for hemophilia with help from a $12 million grant from the National Heart Lung and Blood Institute.

8 Mar 2022

Combating COVID cytokine storm with targeted nucleic acid therapies

Researchers examine current nucleic acid-based treatments against COVID-19 and their methods of action.

6 Mar 2022

Government invests £260 million to support health research and life sciences manufacturing

Patients across the country are set to benefit from cutting-edge medical advances and treatments as the government invests £260 million today (Wednesday 2 March) to support research and development, as well as the manufacturing of new drugs, devices and diagnostics.

2 Mar 2022

Single Use Support brings cell & gene therapy expert Katy Spink on board

Katherine (Katy) Spink has become a member of Single Use Support’s Board of Directors. Her many years of expertise in cell & gene therapy consulting will enrich the companies’ focus on innovating solutions for advanced therapies.

From Single Use Support GmbH 25 Feb 2022

Advanced gene therapy may prevent severe loss of muscle mass in elderly people

Researchers at NTNU have managed to restore muscle function in older mice with muscle loss using advanced gene therapy. The hope is that this method might eventually be used on humans to prevent severe loss of muscle mass.

24 Feb 2022

Research reveals why a common genetic variant worsens outcomes for people with ALS and FTD

A pioneering new study led by UCL and National Institutes of Health (NIH) scientists has revealed, for the first time, why a common genetic variant worsens disease outcomes for people with the devastating adult-onset neurodegenerative diseases amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

24 Feb 2022

Tiny lipid-based nanoparticles carry genetic instructions directly into the lungs

Researchers at the Tufts University School of Engineering are building a reputation for precision targeting in drug delivery.

23 Feb 2022

Gene therapy leads to durable transfusion independence in youngest patients with thalassemia

Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age.

22 Feb 2022

Researchers discover how pre-transplant chemotherapy facilitates the replacement of microglial cells

More than 50,000 bone marrow-derived stem cell transplants are performed worldwide each year to treat a wide range of conditions, including brain diseases.

21 Feb 2022

Drugs that increase fetal hemoglobin levels may help people with sickle cell disease

Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and delayed growth or puberty.

18 Feb 2022

Search for a treatment to stop nerve cell degeneration yields promising results

Researchers at the University of Illinois Chicago found promising results in their search for a treatment to stop nerve cell degeneration that happens in some types of disorders, such as hereditary spastic paraplegia and Parkinson's disease, which can cause significant disability.

17 Feb 2022

Study investigates what causes blood malignancies linked to gene therapy for sickle cell disease

Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored by bluebird bio.

16 Feb 2022

Transformational $104 million gift will help VCU to expand treatment options for liver diseases

Virginia Commonwealth University will radically expand treatment options for liver and liver-related metabolic diseases thanks to a historic, transformational $104 million gift from Richard Todd Stravitz, M.D., and his family's Barbara Brunckhorst Foundation.

15 Feb 2022

Researchers create a comprehensive atlas of cerebrovascular cell types

While neurons and glial cells are by far the most numerous cells in the brain, many other types of cells play important roles. Among those are cerebrovascular cells, which form the blood vessels that deliver oxygen and other nutrients to the brain.