#gene-therapy

#gene-therapy

In a new study published in The New England Journal of Medicine, researchers concluded that a young Hurler syndrome patient who received an adeno-associated virus (AAV) - used to deliver a therapeutic gene to the patient's DNA - later developed a tumor due to the therapy. The patient's illness, Hurler syndrome, stemmed from a mutation that thwarted an enzyme in his body, impacting brain development. A bone marrow transplant was unable to resolve the issue, which ultimately led to the gene therapy.

When mitochondria are exposed to tissue or blood, they lose the electrical gradient across their outer membrane. Mitochondria that lack such a gradient are recognized by a cell's internal machinery as damaged and quickly destroyed. The vast majority of previous studies involved injecting 'naked' mitochondria directly into the bloodstream or tissue sites, but the approach isn't very efficient, so researchers often have to use 'ridiculous' doses of mitochondria.

Oppenheimer frames Ocugen as an emerging gene therapy leader in blinding ocular disorders, with the investment thesis centered squarely on the company's lead asset, OCU400. The firm views a potential 2027 approval as a near-term entry point into what it describes as a 'sizable and poorly-met rare disease market opportunity.' The core appeal is OCU400's gene-agnostic mechanism: unlike the only existing approved retinitis pigmentosa therapy, Luxturna, which targets just one of 100+ RP-associated genes, OCU400 is designed to address the broad RP patient population with a single one-time injection.

As humans, we all want the same thing, a life that's full of good experiences, more time with family, with friends, more time to love, but sometimes genetic illness can cut that short or really, for all of us at some point, our body breaks down. And our bodies are genetic machines. For many diseases, the cause of the disease is a mutation in the genome. Gene therapy is a vision that many have had for decades, more than 50 years.

The biotech company, which is headquartered in Milpitas, turned in a Chapter 7 filing, meaning that it seeks liquidation, rather than reorganization. The document is so sparse that it prompted a request from the court's deputy clerk for more information. But it depicts a company in dire straits: ASC Therapeutics estimates that it has between $100,000 and $500,000 in assets and between $10 million and $50 million in liabilities.

A single multipurpose gene-editing tool can correct several genetic conditions by restoring proteins that have been truncated by disease-causing mutations. The method might one day overcome a key stumbling block faced by gene-editing therapies: the need to design a bespoke treatment for each disease. The new approach, called PERT, combines gene editing with engineered RNA molecules that allow protein synthesis to continue even when a mutation in the DNA tells it to stop prematurely.

GLP-1 is the naturally produced hormone whose function is being mimicked by drugs like semaglutide and tirzepatide, the active ingredients in popular and highly effective weight-loss drugs like Wegovy and Zepbound. As CNN reports, two biotech startups are working on gene therapy treatments that would require, in a best-case scenario, a single injection that would reprogram the body's cells to produce the hormone instead of relying on weekly injections like current drugs.

The revolutionary treatment is administered in just one single dose, with no need for further doses, but it is so sophisticated that it can cost around one million euros. Eliana received it a decade ago and now leads a completely normal life. She gets great grades in school, plays basketball, and has even joined the school choir. It's incredible, her father recounts with emotion.

Research suggests that ancient viral DNA embedded in the human genome is playing a key role in early embryo development. Around 8% of our genome consists of endogenous retrovirus DNA - the remnants of ancient infections, but knowledge of their activity is limited. Now, a team shows that these sequences are required for the correct development of lab-derived embryo analogues, and for the switching on of human-specific genes.

The word skin has come to stand for superficiality and lack of depth, but human skin defies such connotations. For one thing, it is enormous, accounting for roughly 15% of a person's body weight. Skin is also remarkable for its diverse roles: protection against pathogens, temperature regulation and, of course, sensation. Like any part of the body, skin is fallible to disease. But scientists are making great strides in protecting and improving the organ's health.

"It may be one of the first dementias to have a definitive treatment, a cure if you like, a really transformative treatment that allows people to live much longer and much more normal lives..."

The transformation in medicine driven by personalized gene therapies promises cures for many diseases, including cancer, diabetes, and autoimmune conditions, but widespread application may take decades.