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Posts tagged “gene therapy

Reopening avenues for attacking ALS

ALS

ALS

Researchers have found evidence that bone marrow transplantation may one day be beneficial to a subset of patients suffering from amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disorder more commonly known as Lou Gehrig’s disease.

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Some bacterial CRISPRs can snip RNA, too

gene editing

gene editing

You’ve probably seen news stories about the highly lauded, much-discussed genome editing system CRISPR/Cas9. But did you know the system was actually derived from bacteria, which use it to fight off foreign invaders such as viruses? It allows many bacteria to snip and store segments of DNA from an invading virus, which they can then use to “remember” and destroy DNA from similar invaders if they are encountered again. Recent work from a team of researchers including Carnegie’s Devaki Bhaya demonstrates that some bacteria also use the CRISPR/Cas system to snip and recognize segments of RNA, not just DNA.

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Gene therapy treats all muscles in the body in muscular dystrophy dogs

Gene therapy treats all muscles in the body in muscular dystrophy dogs

Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys. Now, a team of University of Missouri researchers have successfully treated dogs with DMD and say that human clinical trials are being planned in the next few years.

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Immune gene prevents Parkinson’s disease and dementia

Immune gene prevents Parkinson's disease and dementia

An estimated seven to ten million people worldwide are living with Parkinson’s disease (PD), which is an incurable and progressive disease of the nervous system affecting movement and cognitive function. More than half of PD patients develop progressive disease showing signs of dementia similar to Alzheimer’s disease.

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Scientists discover new system for human genome editing

Bacteriophages attacking bacteria, TEM

CRISPR systems help bacteria defend against viral attack (shown here).
These systems have been adapted for use as genome editing tools in
human cells.
Image credit goes to: Ami Images/Science Photo Library.

A team including the scientist who first harnessed the revolutionary CRISPR-Cas9 system for mammalian genome editing has now identified a different CRISPR system with the potential for even simpler and more precise genome engineering. In the study researchers describe the unexpected biological features of this new system and demonstrate that it can be engineered to edit the genomes of human cells.

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Researchers resurrect ancient viruses in hopes of improving gene therapy

Researchers resurrect ancient viruses in hopes of improving gene therapy

The Anc80 virus delivered genes to the mouse retina that fluoresce green when expressed. Pictured here, the delivered genes are active in the retina’s color-detecting cells.
Image credit goes to: Livia Carvalho

It sounds like the start of a horror movie, but Harvard Stem Cell Institute (HSCI) researchers at Massachusetts Eye and Ear (MEE) have reconstructed an ancient virus that is highly effective at delivering gene therapies to the liver, muscle, and retina. This discovery could potentially be used to design gene therapies that are not only safer and more potent than therapies currently available, but may also help a greater number of patients.

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