Collaborate Disseminate
CRISPR gene-editing has improved the vision of patients with an inherited form of blindness, according to results of a Phase 1/2 clinical trial. The results not only give new hope to patients with the condition, but show that CRISPR could be put to use… Continue reading CRISPR restores some vision to blind patients in clinical trial
One of the biggest challenges researchers face is making injectable medicines into versions that can be taken orally. Oral medications usually fail because they can’t withstand the harsh environment of the gut, which leads to poor bioavailability – a drug’s ability to be absorbed and exert a therapeutic effect on the body.
Tags: RNA, gene therapy, King’s College London
Continue reading Cow’s milk particles unlock one of medicine’s most challenging puzzles
By silencing the gene responsible for regulating ‘bad’ cholesterol without altering the primary DNA sequence, researchers have shown that it’s possible to use epigenetic editing to treat diseases rather than conventional DNA-breaking gene editing techn… Continue reading ‘Bad’ cholesterol gene silenced without altering the DNA sequence
Researchers have developed a single-dose genetic therapy that can clear protein blockages that cause motor neurone disease, also called amyotrophic lateral sclerosis, and frontotemporal dementia, two incurable neurodegenerative diseases that eventually… Continue reading Single-dose gene therapy may stop deadly brain disorders in their tracks
A breakthrough clinical trial using gene therapy has restored hearing to five children born deaf. After six months, the children were able to recognize speech and hold conversations, raising hopes for wider use in the near future.Continue ReadingCatego… Continue reading Groundbreaking gene therapy trial allows 5 children born deaf to hear
Researchers have discovered that mice possess a natural form of gene therapy, a non-coding RNA that can sidestep genetic mutations. They were able to engineer a programmable version that recognized specific genetic mutations and might be used to treat … Continue reading Mice have innate gene therapy agent to sidestep mutations, study finds
For the first time, the FDA has approved two cell-based gene therapies for treating the rare but potentially fatal sickle cell disease. One is also the first approved therapy to utilize the CRISPR/Cas9 gene editing technology, signifying innovative adv… Continue reading FDA approves first cell-based gene therapies for sickle cell disease
Commensurate with technological improvements and the subsequent rise in genetic research and genetics-based tests and treatments, the term ‘personalized medicine’ is being used more frequently. But what does it mean? This explainer outlines what person… Continue reading Personalized medicine: The pros, cons and concerns
At any given time, as many as four million Americans are suffering through bouts of constipation, which has many causes but can also prove stubborn to relieve and hugely impact daily life.Continue ReadingCategory: Medical, ScienceTags: Gut Bacteria, Ce… Continue reading Probiotic powers genes to relieve constipation, without upsetting gut
The gene-therapy-based treatment called Casgevy was recently approved in the UK, making it the first time that a treatment based on the CRISPR-Cas9 gene editing tool has been authorized for …read more Continue reading First CRISPR-Based Therapies For Sickle Cell Disease and Beta Thalassemia Approved In The UK