gene-therapy

Boston-based biotechnology company Life Biosciences has launched the first-in-human clinical trials of a pioneering “partial cellular reprogramming” technique designed to treat optic nerve damage caused by glaucoma and NAION. Based on previous genetic research, the therapy utilizes a modified virus to deliver three youth-restoring genes to retinal cells, aiming to reverse cellular aging while pre…

The first participant has been treated in a landmark clinical trial of cellular reprogramming, which aims to rejuvenate aging cells

Researchers from Amsterdam UMC, in collaboration with other hospitals, have successfully completed the first-ever Phase 3 study of an in vivo CRISPR therapy.

Revolutionary devices, age rewinding, and cell regeneration: scientists are finding multiple promising approaches for curing blindness, and even for stopping vision loss before it starts.

The first participant has been treated in a landmark clinical trial of cellular reprogramming, which aims to rejuvenate aging cells

An experimental antibody treatment that binds to a protein known as PCDH7 shrank tumors in preclinical models of non-small cell lung cancer (NSCLC), including those resistant to a targeted therapy, a study led by UT Southwestern Medical Center researchers showed. The findings, published in Science Advances, could eventually lead to a new class of drugs […]

A new treatment platform developed by researchers at the University of Texas MD Anderson Cancer Center was able to deliver messenger RNA (mRNA) of the full-length DMD gene into preclinical models of Duchenne muscular dystrophy, successfully restoring the production of an important muscle protein, dystrophin, and dramatically improving muscle strength, endurance and function in vivo. […]

Cancer treatment has improved greatly over the past few decades. Many people with cancer can now undergo surgery, chemotherapy, radiation therapy, or newer targeted treatments and achieve excellent results. Yet one of the biggest challenges remains the same: preventing cancer from returning after treatment appears successful. This problem is especially serious for pancreatic cancer and […] The po…

While non-invasive prenatal testing (NIPT) has revolutionised prenatal diagnostics by allowing the detection of a number of genetic problems in a fetus, it is currently limited and thus misses many genetic causes of abnormalities.

Malcolm Brenner, an internationally recognized physician‑scientist renowned for his leadership in cell and gene therapy, has been tapped to lead the newly formed Houston Methodist Center for Cell and Gene Therapy.

University of Maryland School of Medicine (UMSOM) Researchers have identified a target that may improve the response of CAR T-cell therapy, a treatment for patients with recurrent or difficult to treat blood cancers....

Malcolm Brenner to lead newly-formed Houston Methodist Center for Cell and Gene Therapy

Desperate US parents pay up to $20,000 a session for a procedure scientists say could be bogus Autistic children as young as 18 months old are being injected with human stem cells derived from umbilical cords in unapproved, unproven and potentially harmful “treatments” that scientists warn are proliferating across the US under the active encouragement of the US health secretary, Robert F Kennedy …

Feeling abandoned and overwhelmed, families are turning to controversial new therapies backed by the US health secretary Landyn Holdren is an eight-year-old autistic child who has high support needs and is nonspeaking. His mother, Christy Holdren, says he can be self-harming, slapping his chest, face or head when distressed. Later this month, she will spend $15,000 on an unapproved stem cell trea…

Boston Children's Hospital has been awarded up to $17.25 million to serve as a participating site within the Innovative Medicine and Precision Approaches to Clinical Trials (IMPACT) Network, an international effort funded through Aligning Research to Impact Autism (ARIA).

Doctors say therapy that genetically modifies person’s T-cells could offer cure for chronic autoimmune disease Five lupus patients in England are in remission after being treated with a revolutionary therapy that genetically modifies their own cells, in a medical breakthrough that could offer people a cure, doctors have said. CAR (chimeric antigen receptor) T-cell therapy involves removing a type…

A new treatment platform developed by researchers at The University of Texas MD Anderson Cancer Center was able to deliver messenger RNA (mRNA) of the full-length DMD gene into preclinical models of Duchenne muscular dystrophy, successfully restoring the production of an important muscle protein, dystrophin, and dramatically improving muscle strength, endurance and function in vivo.

RNA therapeutics have emerged as one of the most promising new classes of medicines. Eight small interfering RNA (siRNA) drugs have already been approved worldwide for the treatment of genetic diseases, yet scientists have not fully understood one of the most fundamental steps underlying their function: how Argonaute, the core protein responsible for gene silencing, becomes activated.

An eight-month-old baby from Israel has become the first human ever to receive an experimental gene therapy designed to replace a missing gene responsible for a devastating neurological disease that until now offered no hope of survival.