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.

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

A new Rare Diseases Action Plan for England will be published today (Tuesday 28 February) that will ensure those living with these conditions receive better care and treatment, fairer access to testing and have continued support.

28 Feb 2023

Rapid AAV Titer for Bioprocessing in Gene Therapy

In this article, News-Medical talks to Sartorius about biosensing and bioprocessing in gene therapy, while also discussing the various products Sartorius produces in order to aid in this.

From Sartorius 27 Feb 2023

Safety of a gene therapy to treat alpha 1-antitrypsin deficiency

Researchers report on the safety of a gene therapy to treat the common autosomal recessive hereditary disorder alpha 1-antitrypsin (AAT) deficiency in a new article in the peer-reviewed journal Human Gene Therapy.

22 Feb 2023

UTHSC researchers secure $308,000 grant from Department of Defense for dementia study

Repeated traumatic brain injuries (TBI) in soldiers and military personnel can cause behavioral, neurological, and cognitive effects and lead to dementia.

19 Feb 2023

OHSU researchers confirm first-ever nonhuman primate model of Usher syndrome

Those with Usher Syndrome -- the leading hereditary cause for simultaneous deafness and blindness, for which there is no treatment -- may have a new reason for hope now that researchers have confirmed the first-ever nonhuman primate model of their disease.

13 Feb 2023

Cerba Research and Teddy Lab sign Memorandum of Understanding on entering into a discussion for Joint Venture

Cerba Research announced today a Memorandum of Understanding with Teddy Clinical Research Laboratory (Wuxi, China), a preeminent central lab provider covering mainland China, to lay the foundation for an in-depth discussion towards the creation of a Joint Venture.

From Cerba Research 3 Feb 2023

The human body develops strategies to prevent the immune response from overshooting its mark

The signaling molecules of the immune system should trigger a response only where necessary. To prevent a life-threatening spread to the rest of the body, connective tissue can absorb these molecules like a sponge.

3 Feb 2023

New 'cutting edge' hemophilia treatment is a victory for patients, Tulane doctor says

A new treatment that helps people with hemophilia A maintain higher levels of a crucial blood clotting factor with fewer treatments is a victory for patients, according to a new editorial in The New England Journal of Medicine this week by Dr. Cindy Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University School of Medicine.

27 Jan 2023

AI tool promises to accelerate the development of gene therapies on a large scale

An artificial intelligence (AI) program may enable the first simple production of customizable proteins called zinc fingers to treat diseases by turning genes on and off.

26 Jan 2023

Caron Receives Patents for Humanity COVID-19 Program Award from the United States Patent and Trademark Office

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26 Jan 2023

Mitochondrial dysfunction and endothelial impairment linked to many cardiovascular diseases

Many cardiovascular diseases, such as atherosclerosis, or 'hardening of the arteries,' correlate to mitochondrial dysfunction and endothelial impairment in the tissues of the heart and blood vessels.

23 Jan 2023

Healthy gene found in centenarians can reverse the decline of heart performance in older people

An anti-aging gene discovered in a population of centenarians has been shown to rewind the heart's biological age by 10 years.

23 Jan 2023

Therapeutic small molecule can restore visual functions after optic nerve injury

Traumatic injury to the brain, spinal cord and optic nerve in the central nervous system (CNS) are the leading cause of disability and the second leading cause of death worldwide.

20 Jan 2023

Gene editing could offer a promising solution for treating patients after a heart attack

Editing a gene that prompts a cascade of damage after a heart attack appeared to reverse this inevitable course in mice, leaving their hearts remarkably unharmed, a new study by UT Southwestern scientists showed.

20 Jan 2023

Partial genetic reprogramming might extend lifespan and reverse aging in old mice

Researchers performed age-reversing experiments, in vivo and in vitro, in progeroid mice using gene therapy.

19 Jan 2023

Using the power of light to transform analytical workflows in the life sciences

In this interview, we speak to Svea Cheeseman at Refeyn about their SamuxMP products used for the analysis of adeno-associated viruses (AAV) capsids.

From Refeyn 17 Jan 2023

TTP introduces Cellular Origins, a TTP Company focussed on delivering scalable manufacture of cell and gene therapies

New company created to enable commercial manufacture of new cell and gene therapies.

From TTP plc 17 Jan 2023

FUJIFILM Irvine Scientific launches HEK293 Feed, specifically designed for viral vector production

FUJIFILM Irvine Scientific, Inc., a world leader in the development and manufacture of serum-free and chemically defined cell culture media for bioproduction and cell therapy manufacturing, today announced the launch of its BalanCD HEK293 Viral Feed.

11 Jan 2023

Vector BioPharma and iBET collaborate to develop high-throughput analytical methods and scalable processes for gene delivery

Collaboration set to develop and implement high-throughput analytical methods in support of Vector BioPharma’s CMC (chemistry, manufacturing and controls) development.

From Vector BioPharma 10 Jan 2023

Clinically relevant biomarker strategies in drug development

In this interview, NewsMedical speaks with Dr. Justin Devine and Dr. Nicholas Woudberg about biomarkers, their importance for drug development as well as the potential they have when it comes to machine learning.

From Synexa Life Sciences BV 9 Jan 2023