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.

Innovative gene therapy to cure multiple eye diseases

The future treatment of individuals with various eye diseases could benefit immensely from a potential new gene therapy strategy, according to Trinity University researchers.

31 Jan 2023

Anti-aging gene found in centenarians can rewind the heart's biological age by 10 years

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

31 Jan 2023

Deep-learning model for zinc finger design allows reprogramming of human transcription factors

Researchers at the University of Toronto and New York University have developed a novel technology that can engineer proteins to target any stretch of DNA in the human genome, opening a door toward gene therapies for a broader range of health conditions.

27 Jan 2023

Gene editing made faster than ever before using AI

The first simple production of customizable proteins known as zinc fingers to treat diseases by turning genes on and off might be enabled with the help of an artificial intelligence (AI) program.

From New York University (NYU) 27 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

Scientist honored for his contribution on ALS research

The Drs Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing was given to Clive Svendsen, PhD, a pioneer in regenerative medicine, for his significant contributions to the field of amyotrophic lateral sclerosis (AL) at Massachusetts General Hospital’s Sean M. Healey & AMG Center for ALS.

12 Dec 2022

Newly developed tool helps snip out faulty genes in a safe manner

Scientists from the Massachusetts Institute of Technology (MIT) have come up with a new tool that has the potential to detach faulty genes and exchange them with new ones in a highly safe and effective way.

From Massachusetts Institute of Technology (MIT) 25 Nov 2022

Tissue nanotransfection technology could help treat traumatic muscle loss

Technology developed by researchers at the Indiana University School of Medicine that can change skin tissue into blood vessels and nerve cells has also shown promise as a treatment for traumatic muscle loss.

10 Nov 2022

New tool advances potential means to deliver gene therapy

Researchers from Johns Hopkins Medicine claim to have successfully “slid” genetic instructions into a cell and produced essential proteins that were lacking from those cells using a cell’s normal process for creating proteins.

1 Nov 2022

Pioneering gene editing approach could lead to new treatments for CTLA-4 insufficiency

A fault in cells that form a key part of the immune system can be repaired with a pioneering gene editing technique, finds new research demonstrated in human cells and mice, led by UCL scientists.

From University College London 27 Oct 2022

A more efficient way to deliver gene therapies to the CNS

For gene therapy, the blood-brain barrier (BBB) poses a formidable obstacle. The BBB, which is made up of cells that are closely packed together, prevents poisons and pathogens from accessing brain tissue while also blocking potential treatments for diseases that impact the CNS.

12 Oct 2022

Adults with congenital blindness experience striking recoveries of night vision after gene therapy

Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.

11 Oct 2022

Astrea Bioseparations introduces Nereus LentiHERO, a fit-for-purpose solution for lentiviral vector purification

Astrea Bioseparations (“Astrea Bio”), a leading provider of novel purification and separation tools supporting the development of next-generation therapeutics, has announced today the commercial launch of its Nereus LentiHERO™ lentiviral vector (LVV) purification technology.

29 Sep 2022

Study highlights retrotransposition in CRISPR gene therapy clinical trials

Worldwide clinical studies for gene therapy utilizing CRISPR/Cas9 gene editing are now underway for a number of disorders. A study from Boston Children’s Hospital that was released in Nature Communications on June 27th, 2022, alerts readers to a possible CRISPR editing risk that has not yet been identified.

8 Jul 2022

Gene Therapy News and Research

Innovative gene therapy to cure multiple eye diseases

Anti-aging gene found in centenarians can rewind the heart's biological age by 10 years

Deep-learning model for zinc finger design allows reprogramming of human transcription factors

Gene editing made faster than ever before using AI

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

Scientist honored for his contribution on ALS research

Newly developed tool helps snip out faulty genes in a safe manner

Tissue nanotransfection technology could help treat traumatic muscle loss

New tool advances potential means to deliver gene therapy

Pioneering gene editing approach could lead to new treatments for CTLA-4 insufficiency

A more efficient way to deliver gene therapies to the CNS

Adults with congenital blindness experience striking recoveries of night vision after gene therapy

Astrea Bioseparations introduces Nereus LentiHERO, a fit-for-purpose solution for lentiviral vector purification

Study highlights retrotransposition in CRISPR gene therapy clinical trials

Researchers discover a safe strategy to correct genetic defects

Researchers reveal that new compact version of Cas7-11 enzyme improves RNA editing

Scientists develop powerful tools using CRISPR-Cas9 to repair genetic diseases

Study shows polymersome can effectively deliver PARP1 siRNA to treat breast cancer

Rapidly growing nanotechnology firm launches new identity as MIP Discovery

Research reveals that pain-blocking neurotransmitters offer long-term relief in mice

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