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.

Chemical modification in RNA molecules may offer protection against liver disease

A chemical modification that occurs in some RNA molecules as they carry genetic instructions from DNA to cells' protein-making machinery may offer protection against non-alcoholic fatty liver, a condition that results from a build-up of fat in the liver and can lead to advanced liver disease, according to a new study by UCLA researchers.

19 Jul 2021

Gene therapy delivered into the brain may be safe, effective in treating rare neurological disorder

A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency.

16 Jul 2021

Researchers study the role of subnuclear NSrp70 in T cell-mediated adaptive immunity

T cells are components of the body’s immune system. The ability of T cells to recognize diverse pathogens and mount a defense against them stems from the way proteins are spliced during their development and maturation.

15 Jul 2021

Yale School of Public Health and Tevogen Bio partner to expand school's regulatory sciences program

The Yale School of Public Health is partnering with Tevogen Bio, a clinical stage biotechnology company, on a major expansion of the school's regulatory sciences program.

14 Jul 2021

Curia to Acquire US-based LakePharma, Expanding End-to-End R&D Capabilities and Scale for Biologics

Curia today announced it has entered into a definitive agreement to acquire LakePharma Inc.

From Curia 14 Jul 2021

Gene therapy shows promise in children with devastating genetic disorder

Children with a devastating genetic disorder characterized by severe motor disability and developmental delay have experienced sometimes dramatic improvements in a gene therapy trial launched at UCSF Benioff Children's Hospitals.

12 Jul 2021

Novel method of gene therapy promises to transform the treatment of rare genetic disorder

A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities.

12 Jul 2021

Study: Gene editing therapy for early-stage Huntington’s disease may have long-lasting benefits

In a new study on mice, Johns Hopkins Medicine researchers report that using MRI scans to measure blood volume in the brain can serve as a noninvasive way to potentially track the progress of gene editing therapies for early-stage Huntington's disease, a neurodegenerative disorder that attacks brain cells.

7 Jul 2021

Nanotechnology and the fight against COVID-19

In a review paper published in the open-access MDPI journal Viruses, a team of researchers explored the potential of nanotechnology to alleviate the current and future coronavirus burden. The team focused on its potential role in the detection, diagnosis, protection, and treatment of SARS-CoV-2 infection.

29 Jun 2021

Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Genethon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of the Liver) annual International Liver Congress on June 26.

28 Jun 2021

RDM Positive Impact Foundation funds $1.25 million research project to study SYNGAP1

The RDM Positive Impact Foundation is funding an ambitious $1.25 million research project at the UC Davis MIND Institute to study SYNGAP1.

22 Jun 2021

Researchers design a smart device that delivers medications to prevent HIV transmission

Researchers have designed a device that delivers two medications that help stop HIV transmission.

18 Jun 2021

Repressing a protein that facilitates DNA repair to potentiate chemotherapy

Chemotherapy kills tumor cells by causing damage to them. One of the most effective ways of causing damage is to prevent the two DNA strands from separating so that the cellular machinery cannot read the instructions written in the genes.

15 Jun 2021

New approach to gene therapy uses common pain reliever to fight against genetic diseases

Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission.

14 Jun 2021

Researchers identify a common mechanism underlying diverse brain disorders

Researchers at Vanderbilt University Medical Center have identified a common mechanism underlying a spectrum of epilepsy syndromes and neurodevelopmental disorders, including autism, that are caused by variations in a gene encoding a vital transporter protein in the brain.

9 Jun 2021

Experimental monoclonal antibody could be a new weapon against HIV epidemic

An experimental, lab-made antibody can completely prevent nonhuman primates from being infected with the monkey form of HIV, new research published in Nature Communications shows.

7 Jun 2021

Gene therapy may be a safe and effective single-dose treatment for soft bone disease

A preclinical study led by scientists at Sanford Burnham Prebys has established that AAV8-TNAP-D10--a gene therapy that replaces a key enzyme found in bone--may be a safe and effective single-dose treatment for hypophosphatasia (HPP).

7 Jun 2021

Researchers create cellular and spatial “atlas” of the choroid plexus during different life stages

Once viewed merely as a producer of the cerebrospinal fluid (CSF) bathing the brain and spinal cord, the choroid plexus is now known to be a key player in brain development and immunity.

7 Jun 2021

A lack of oversight for unproven stem cell interventions is a global public health problem

Authors are urging the WHO to establish an Exert Advisory Committee on Regenerative Medicine to provide harmony to the national regulations on the SCI industry.

4 Jun 2021

Researchers develop proof-of-concept treatment for blood disorders

Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin.

1 Jun 2021

Gene Therapy News and Research

Further Reading

Chemical modification in RNA molecules may offer protection against liver disease

Gene therapy delivered into the brain may be safe, effective in treating rare neurological disorder

Researchers study the role of subnuclear NSrp70 in T cell-mediated adaptive immunity

Yale School of Public Health and Tevogen Bio partner to expand school's regulatory sciences program

Curia to Acquire US-based LakePharma, Expanding End-to-End R&D Capabilities and Scale for Biologics

Gene therapy shows promise in children with devastating genetic disorder

Novel method of gene therapy promises to transform the treatment of rare genetic disorder

Study: Gene editing therapy for early-stage Huntington’s disease may have long-lasting benefits

Nanotechnology and the fight against COVID-19

Preliminary results of AAV-mediated gene therapy for Crigler Najjar syndrome

RDM Positive Impact Foundation funds $1.25 million research project to study SYNGAP1

Researchers design a smart device that delivers medications to prevent HIV transmission

Repressing a protein that facilitates DNA repair to potentiate chemotherapy

New approach to gene therapy uses common pain reliever to fight against genetic diseases

Researchers identify a common mechanism underlying diverse brain disorders

Experimental monoclonal antibody could be a new weapon against HIV epidemic

Gene therapy may be a safe and effective single-dose treatment for soft bone disease

Researchers create cellular and spatial “atlas” of the choroid plexus during different life stages

A lack of oversight for unproven stem cell interventions is a global public health problem

Researchers develop proof-of-concept treatment for blood disorders

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