‘Bad’ cholesterol gene silenced without altering the DNA sequence

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

Single-dose gene therapy may stop deadly brain disorders in their tracks

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

Groundbreaking gene therapy trial allows 5 children born deaf to hear

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

Mice have innate gene therapy agent to sidestep mutations, study finds

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

FDA approves first cell-based gene therapies for sickle cell disease

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

Probiotic powers genes to relieve constipation, without upsetting gut

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

First CRISPR-Based Therapies For Sickle Cell Disease and Beta Thalassemia Approved In The UK

A giemsa stained blood smear from a person with beta thalassemia (Credit: Dr Graham Beards, Wikimedia Commons)

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

Thousands of Fanzor DNA cutters that may outperform CRISPR identified

Following on from the discovery of programmable DNA-cutting enzymes known as Fanzors, scientists have found that a diverse range of species possess these genetic ‘scissors’, which presents a massive opportunity in the development of new medicines, gene… Continue reading Thousands of Fanzor DNA cutters that may outperform CRISPR identified

Targeted gene therapy helps completely paralyzed mice walk again

A complete spinal cord injury results, tragically, in total paralysis of all limbs and muscles below the injury site. But now, scientists at EPFL have demonstrated in mice a new gene therapy that can regenerate nerves and restore the ability to walk.Co… Continue reading Targeted gene therapy helps completely paralyzed mice walk again