Lifeboat Foundation

He is the first patient to receive an experimental gene therapy as part of a clinical trial. Earlier this week, Sangamo Therapeutics injected Madeux with viruses containing a package of gene-editing material, according to the AP. The hope is that these viruses will enter Madeux’s cells, specifically liver cells, inject the missing gene at the right place in his DNA. Only about 1% of the liver’s cells need to be fixed, and give his liver the ability to produce the enzyme he has been missing all his life.

Brian Madeux’s life hasn’t been easy. So far, he’s had 26 operations to fix problems in everything from hernias to eyes. He has a rare disease called Hunter syndrome, which is caused by the lack of a gene that’s used to produce an enzyme that breaks down certain carbohydrates. As a result, the carbohydrates build up in his body’s cells causing all sorts of problems.

Continue reading “Doctors are gene editing inside the body of a living human for the first time” »

The success of a novel gene therapy for blindness caused by a genetic mutation paves the way for gene therapies which treat other forms of blindness as well as genetic therapies which treat other diseases.

After being treated with a novel gene therapy for blindness, patients who had lost their sight due to a genetic retinal disease could see well enough to navigate a maze, according to research presented today at the 2017 Annual Meeting of the American Academy of Ophthalmology (AAO 2017).

Novel Gene Therapy for Blindness

Continue reading “Gene Therapy for Blindness May Soon Be Reality” »

“Scientists are hopeful that the approach could offer new ways to understand – and even one day tackle – certain human genetic diseases by correcting mutations in a patient’s body.”

Fresh DNA base editing breakthrough brings hope of potential treatment for huge number of diseases that arise as a result of a single genetic ‘misspelling’.

The CRISPR-Cas9 system has revolutionised gene-editing, but cutting DNA isn’t all it can do. From turning gene expression on and off to fluorescently tagging particular sequences, this animation explores some of the exciting possibilities of CRISPR.

Download a poster on ‘The expanding CRISPR toolbox’ here: https://www.nature.com/posters/crisprtoolbox

Continue reading “Gene editing and beyond” »

‘It’s another tool in the toolbox’

Over just a few short years the CRISPR gene-editing technique has revolutionized science, affecting everything from medicine to agriculture. Two new breakthrough studies have just been published describing dual methods that make the process more precise and efficient paving the way for scientists to safely alter DNA mutations that cause thousands of different human diseases.

CRISPR is conventionally a cut-and-paste tool allowing scientists to chop out unwanted strands of DNA and insert new genes, but a large volume of human diseases are caused by a single point mutation somewhere in a person’s DNA. Up until now scientists have not been able to simply and directly erase or rewrite these single mutations in living human cells.

Our human genome consists of 3 billion base pairs made up of chemical units referred to by the letters A, C, G and There are 50,000 known genetic mutations that are linked to disease in humans and 32,000 of these are single point mutations. Half of those single point mutations have been identified as a G-C pair that has mutated into an A-T pair.

Just like how letters are strung together to form words, our DNA is also strung together by letters to encode proteins. The genetic alphabet contains only 4 natural letters — A, C, G and T, which hold the blueprint for the production of proteins that make our bodies work. Now, researchers from the Institute of Bioengineering and Nanotechnology (IBN) of the Agency for Science, Technology and Research (A*STAR) have created a DNA technology with two new genetic letters that could better detect infectious diseases, such as dengue and Zika.

Genetic alphabet expansion technology is the introduction of artificial base pairs into DNA. The existing four genetic letters are naturally bound together in base pairs of A-T and G-C. These specific base pair formations are essential in DNA replication, which occurs in all living organisms. It is the process by which a DNA molecule is duplicated to produce two identical molecules.

“The expansion of the genetic alphabet is a significant scientific achievement. It sheds insights into DNA’s natural replication mechanism, which will help us to design unique DNA molecules and technologies. For example, our technology can be used to create novel diagnostics and therapeutic agents with superior efficacy,” said IBN Executive Director Professor Jackie Ying.

There are multiple software options that analyze your genetic data and make recommendations on which supplements you should be taking.

Scientists are altering a powerful gene-editing technology in hopes of one day fighting diseases without making permanent changes to people’s DNA.

The trick: Edit RNA instead, the messenger that carries a gene’s instructions.

“If you edit RNA, you can have a reversible therapy,” important in case of side effects, said Feng Zhang of the Broad Institute of MIT and Harvard, a gene-editing pioneer whose team reported the new twist Wednesday in the journal Science.