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 genetically-targeted MND therapy could be a turning point for patient care

Scientists believe a new genetically-targeted therapy to treat motor neurone disease (MND) could be a turning point for patient care, after the results of a Phase 3 clinical trial showed significant physical benefits for patients after 12 months.

21 Sep 2022

Study identifies a promising target for polycystic kidney disease treatment

Blocking the inhibition of PKD1 and PKD2 gene expression by deleting a binding site for microRNAs hindered the formation and growth of kidney cysts in autosomal dominant polycystic kidney disease (ADPKD) models, UT Southwestern researchers reported.

16 Sep 2022

Treating the cerebrospinal fluid to protect the brain from chemotherapy's toxic effects

Up to three quarters of patients receiving cancer chemotherapy suffer from "chemo brain" - a long-term side effect that makes it harder to remember things, maintain attention, and learn new information. When it strikes children, whose brains are still developing, their schoolwork and self-esteem may suffer.

13 Sep 2022

New gene therapy approach could help prevent blindness in children with Leber congenital amaurosis

Researchers from the National Eye Institute have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood.

8 Sep 2022

Combined stem cell and gene therapy shows promise to treat patients with ALS

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier.

5 Sep 2022

Alternative approaches to functional and phenotypic assays using multi-parameter flow cytometry

Synexa is an industry-leading biomarker and large molecule bio analytic research service provider. In this interview, NewsMedical speaks with Natalie Strickland, a principal scientist at Synexa, about alternative approaches to flow cytometry.

From Synexa Life Sciences 5 Sep 2022

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

Researchers receive grant to develop purification membranes for large-scale manufacturing of viral vectors

With a $6 million grant from the National Science Foundation, researchers at the University of Arkansas and two partner institutions will develop purification membranes for future large-scale manufacturing of viral vectors and virus-like particles.

19 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