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.

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

Researchers discover a safe strategy to correct genetic defects

Healing debilitating genetic diseases is one of modern medicine’s most difficult challenges.

5 Jul 2022

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

Cas7-11, the first CRISPR enzyme that can make precise, directed cuts to strands of RNA without hurting cells, was identified and described last year by researchers at MIT’s McGovern Institute for Brain Research.

From Massachusetts Institute of Technology (MIT) 2 Jun 2022

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

CRISPR has marked the beginning of a new era in genomic medicine. The popular CRISPR-Cas9 has generated plenty of potent methods for curing genetic diseases.

30 May 2022

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

Small interfering RNAs, or siRNAs, have the potential to cure tumors by targeting knocking down oncogenes that drive tumor development while avoiding the damage associated with chemotherapy.

25 May 2022

Rapidly growing nanotechnology firm launches new identity as MIP Discovery

MIP Diagnostics, a leading UK nanotechnology firm has today rebranded to MIP Discovery as the business continues its expansion within the healthcare sector.

From MIP Discovery 19 May 2022

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

An international team of researchers found that a gene therapy that prevents targeted nerve cell signaling substantially lower neuropathic pain with no detectable side effects.

From UC San Diego 12 May 2022

Scientists reveal how autism-linked genetic mutation disrupts neural development using mini-brains

In a study published May 02, 2022 in Nature Communications, scientists at University of California San Diego School of Medicine used human brain organoids to reveal how a genetic mutation associated with a profound form of autism disrupts neural development.

From UC San Diego 3 May 2022