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.

Novel gene-based therapies for sickle cell disease rely on finding the right mouse for the job

Sickle cell disease is an extremely debilitating condition that affects up to 40% of the population in African countries, with patients suffering episodes of excruciating pain, organ damage and reduced life-expectancy.

7 Jul 2022

The Life Sciences; Where are we now, and where are we going?

As part of our SLAS Europe 2022 coverage, we spoke to Gerrit Gutzke and Michael Bussmann from Azenta Life Sciences about the products they offer for life sciences research.

From Society for Laboratory Automation and Screening (SLAS) 7 Jul 2022

New, safer CRISPR approach may help correct genetic defects in the future

Curing debilitating genetic diseases is one of the great challenges of modern medicine. During the past decade, development of CRISPR technologies and advancements in genetics research brought new hope for patients and their families, although the safety of these new methods is still of significant concern.

1 Jul 2022

Cancer drug helped delay progression of Duchenne muscular dystrophy in mice

Researchers at UBC’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.

1 Jul 2022

HKUMed researchers discover more efficient CRISPR-Cas9 variants for gene therapy applications

A research team from the LKS Faculty of Medicine, The University of Hong Kong discovered more efficient CRISPR-Cas9 variants that could be useful for gene therapy applications.

30 Jun 2022

Existing cancer drug shows promise for treating patients with muscular dystrophy

Researchers at the University of British Columbia's School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.

29 Jun 2022

Cellular "barcoding" in mice unravels the origins of blood

The origins of our blood may not be quite what we thought. Using cellular "barcoding" in mice, a groundbreaking study finds that blood cells originate not from one type of mother cell, but two, with potential implications for blood cancers, bone marrow transplant, and immunology. Fernando Camargo, PhD, of the Stem Cell Program at Boston Children's Hospital led the study, published in Nature on June 15.

15 Jun 2022

Scientists discover novel species of betapolyomavirus in Kenya

A new study published as a pre-proof in the journal Virologica Sinica, is follow-up research to a previous study on the discovery of novel rodent viruses of various RNA viral families in Kenya.

12 Jun 2022

New fund could enable early access to life-saving treatments for NHS patients in England

NNHS patients in England will have early access to potentially life-saving and cutting-edge treatments thanks to a new fund which has been launched today.

6 Jun 2022

Gene therapy for degenerative retinal disease has no safety concerns but fails to improve vision loss

A 28-patient phase 1 gene therapy clinical trial for the degenerative retinal disease Leber hereditary optic neuropathy (LHON) found no significant safety concerns; however, treatment failed to improve or slow vision loss, with even the highest dose. LHON affects the optic nerve, which carries visual signals from the light-sensing retina to the brain.

1 Jun 2022

Understanding transposons provides a new starting point to generate more powerful gene editing tools

CRISPR has ushered in the era of genomic medicine. A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases.

27 May 2022

Biodegradable, non-ionic polymeric nanovesicles efficiently encapsulate and deliver siRNA

Small interfering RNAs, or siRNAs, hold promise to treat tumors, through their ability to specifically knock down oncogenes that promote tumor growth, without the toxicity that accompanies chemotherapy. However, the siRNAs need a delivery vehicle to protect them from degradation and clearance on their journey through the bloodstream to the cancer tumor.

25 May 2022

Discoveries may lead to new ways to treat patients with Allan-Herndon-Dudley syndrome

Two recent discoveries co-led by scientists at Cedars-Sinai may help lead to new ways to treat patients with Allan-Herndon-Dudley syndrome (AHDS), a brain development disorder that causes severe intellectual disability and problems with movement.

20 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

Mice live longer with nasal gene therapy

In a recent study published in the Proceedings of the National Academy of Sciences journal, researchers assessed the efficiency of a novel intranasal and injectable therapy in improving life expectancy.

17 May 2022

Study highlights the economic burden affecting individuals with sickle cell disease

In a study published today in Blood Advances, researchers found that privately insured individuals with sickle cell disease (SCD) spend approximately $1.7 million on disease-related medical expenses over their lifetime.

16 May 2022

Postnatal gene therapy may be able to prevent or reverse deadly effects of Pitt-Hopkins syndrome

UNC School of Medicine Scientists have shown for the first time that postnatal gene therapy may be able to prevent or reverse many deleterious effects of a rare genetic disorder called Pitt-Hopkins syndrome.

10 May 2022

Gene therapy effectively reduces neuropathic pain in mice with spinal cord injuries

An international team of researchers, led by scientists at University of California San Diego School of Medicine, report that a gene therapy that inhibits targeted nerve cell signaling effectively reduced neuropathic pain with no detectable side effects in mice with spinal cord or peripheral nerve injuries.

9 May 2022

Study uses brain organoids to reveal how autism-linked genetic mutation disrupts neural development

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.

2 May 2022

Better understanding of hydrocephalus could lead to improved treatments

Hydrocephalus, or "water on the brain," occurs when the cerebral ventricles - four interconnected cavities of the brain that are filled with cerebrospinal fluid - become enlarged, but its cause is unknown in many cases.

18 Apr 2022