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.

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

SynGAP Research Fund awards $180,000 grant to the University of Edinburgh Medical School's Patrick Wild Centre & Centre for Discovery Brain Sciences

SynGAP Research Fund (SRF), a 501(c)(3) public charity whose mission is to improve the quality of life for SYNGAP1 patients through the research and development of treatments, therapies and support systems, along with SynGAP Research Fund UK, today announced they have awarded a $180,000 grant to the University of Edinburgh Medical School's Patrick Wild Centre & Centre for Discovery Brain Sciences to advance the science around gene transfer to correct SYNGAP1 haploinsufficiency.

5 Jan 2023

'Stem cell plasters' could revolutionize treatment for children with congenital heart disease

Researchers at the University of Bristol funded by the British Heart Foundation (BHF) have developed 'stem cell plasters' to revolutionize the way surgeons treat children living with congenital heart disease, so they don't need as many open-heart operations.

20 Dec 2022

Many genetic disorders respond well to specialized gene therapy

When the normal DNA sequence in an individual changes or mutates it can cause disease. The mutation may involve multiple genes and may be caused by environmental factors, or there may be chromosomal defects that are harmful at birth, in infancy or environment at a later stage.

12 Dec 2022

GOSH delivers world-first base-edited cell therapy for a patient with relapsed T cell leukemia

A patient with relapsed T cell leukemia has been given base-edited T-cells in a world-first use of a base-edited cell therapy, in a 'bench-to-bedside' collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).

12 Dec 2022

Researchers seek to reduce Chikungunya virus-caused joint pain with new drug

Oregon Health & Science University researchers and partners are developing what could become the first-ever treatment against the debilitating joint pain that can last months or years after becoming infected with the emerging Chikungunya virus.

9 Dec 2022

Study looks at the possibility of promoting hair cell regeneration using cell reprograming

About 430 million people around the world experience disabling hearing loss. In the United States, approximately 37.5 million adults report some trouble hearing. Hearing loss can happen when any part of the ear or the nerves that carry information on sounds to the brain do not work in the usual way.

8 Dec 2022

Liver cancer study could help improve the safety of certain gene therapies for hemophilia

Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases.

7 Dec 2022

Shenandoah Biotechnology introduces expanded range of CTGrade recombinant proteins manufactured following cGMP practices

Shenandoah Biotechnology Inc., a FUJIFILM Irvine Scientific company and leading manufacturer of recombinant proteins, today announced it has expanded its CTGrade portfolio of cytokines and growth factors for cell and gene therapies manufactured following cGMP guidelines.

From FUJIFILM Irvine Scientific 7 Dec 2022