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.

Understanding how microglia change their states to adapt to different areas of the brain

In this interview, News Medical speaks to Jeffrey Stogsdill, Ph.D., about his latest research exploring how microglia change their state to adapt to different areas of the brain.

2 Sep 2022

New approach using CRISPR can engineer massive quantities of cells for therapeutic applications

A new variation of the CRISPR-Cas9 gene editing system makes it easier to re-engineer massive quantities of cells for therapeutic applications.

25 Aug 2022

Next generation mRNA vaccines exhibit excellent protective and therapeutic effects

Researchers around the world have been working for years on developing vaccines against different types of cancer, but without much success. Now, Tufts School of Engineering researchers think they have found one that does work. They have devised a method of targeting cancer in mice with a vaccine that is so strong and precise, it eliminates tumors and even prevents their recurrence.

16 Aug 2022

Higher doses of CAR-T therapy achieve better survival rates for young patients with hard-to-treat B-ALL

Young people who received doses of tisagenlecleucel, a chimeric antigen receptor T cell (CAR-T) therapy, at the higher end of the FDA-approved dosing range had significantly better survival rates at one year compared with those who received lower doses within this range, according to research published today in Blood Advances.

8 Aug 2022

Insights into the products and services of Azenta

This interview talks to Andre Kotze about the different services and products available at Azenta.

From Azenta Life Sciences 8 Aug 2022

Terminally exhausted T cells may remain competent for transcription, researchers say

During a battle with cancer, T cells can become exhausted and are no longer able to function properly. The early phase of exhaustion can sometimes be reversed with immunotherapy drugs, but once T cells become too exhausted, it had been thought that this state was irreversible. However, new insights from University of Pittsburgh and UPMC researchers suggest that even the most fatigued T cells can be revived.

8 Aug 2022

Gene therapy approach delays disease onset in humanized mouse models of familial ALS

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the progressive loss of motor neurons in the brain and spinal cord responsible for voluntary movements and muscle control.

2 Aug 2022

New DNA repair vehicle used to fix a common genetic cause of hereditary kidney disease

Genetic mutations which cause a debilitating hereditary kidney disease affecting children and young adults have been fixed in patient-derived kidney cells using a potentially game-changing DNA repair-kit. The advance developed by University of Bristol scientists is published in Nucleic Acids Research.

29 Jul 2022

Ultra-precise gene therapy technologies could edit or silence faulty genes causing fatal heart diseases

An injectable cure for inherited heart muscle conditions that can kill young people in the prime of their lives could be available within a few years, after an international team of researchers were announced as the winners of the British Heart Foundation's Big Beat Challenge.

29 Jul 2022

Mesoporous silica nanoparticles: Possibilities in therapeutic development

In this interview, News-Medical talks to Philippe Riviere, Senior Scientist at NanoComposix (now a Fortis Life Sciences company) about mesoporous silica nanoparticles and the possibilities and opportunities they provide to drug delivery, vaccine development and more.

From nanoComposix 28 Jul 2022

Scientists receive $3.9 million NIH grant to eradicate HIV from the brain

Finding a cure for HIV has been extremely difficult, in part because the virus hides from antiretroviral treatments in one of the hardest places to reach: the brain.

27 Jul 2022

Insights into molecular folding of AAT protein will help develop new therapies for alpha-1 antitrypsin deficiency

In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School recently announced that they have mapped the expression and maturation of the protein alpha-1 antitrypsin (AAT) with unprecedented clarity.

25 Jul 2022

Parents become drug developers to save their children’s lives

Maggie Carmichael wasn't developing like other kids. As a toddler, she wasn't walking and had a limited vocabulary for her age.

21 Jul 2022

Novel gene-based therapies for sickle cell disease rely on finding the right mouse for the job

Sickle cell disease is an extremely debilitating condition that affects up to 40% of the population in African countries, with patients suffering episodes of excruciating pain, organ damage and reduced life-expectancy.

7 Jul 2022

New, safer CRISPR approach may help correct genetic defects in the future

Curing debilitating genetic diseases is one of the great challenges of modern medicine. During the past decade, development of CRISPR technologies and advancements in genetics research brought new hope for patients and their families, although the safety of these new methods is still of significant concern.

1 Jul 2022

Cancer drug helped delay progression of Duchenne muscular dystrophy in mice

Researchers at UBC’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.

1 Jul 2022

HKUMed researchers discover more efficient CRISPR-Cas9 variants for gene therapy applications

A research team from the LKS Faculty of Medicine, The University of Hong Kong discovered more efficient CRISPR-Cas9 variants that could be useful for gene therapy applications.

30 Jun 2022

Existing cancer drug shows promise for treating patients with muscular dystrophy

Researchers at the University of British Columbia's School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.

29 Jun 2022

Cellular "barcoding" in mice unravels the origins of blood

The origins of our blood may not be quite what we thought. Using cellular "barcoding" in mice, a groundbreaking study finds that blood cells originate not from one type of mother cell, but two, with potential implications for blood cancers, bone marrow transplant, and immunology. Fernando Camargo, PhD, of the Stem Cell Program at Boston Children's Hospital led the study, published in Nature on June 15.

15 Jun 2022

Scientists discover novel species of betapolyomavirus in Kenya

A new study published as a pre-proof in the journal Virologica Sinica, is follow-up research to a previous study on the discovery of novel rodent viruses of various RNA viral families in Kenya.

12 Jun 2022