Gene Editing News and Research

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UCLA-developed nanospears could make gene therapies safer, faster and more cost-effective

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called "nanospears" for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.

15 Mar 2018

Researchers uncover link between Parkinson’s disease mutations and plaque formation in the brain

Working with lab-grown human brain cells, Johns Hopkins researchers report they have uncovered a much sought-after connection between one of the most common genetic mutations in Parkinson's disease and the formation of fatty plaques in the brain thought to contribute to the destruction of motor neurons that characterize the disease.

15 Mar 2018

NIH-funded researchers use CRISPR to rapidly find new genetic suspects behind ALS, FTD

NIH-funded researchers at Stanford University used the gene editing tool CRISPR-Cas9 to rapidly identify genes in the human genome that might modify the severity of amyotrophic lateral sclerosis and frontotemporal dementia caused by mutations in a gene called C9orf72.

13 Mar 2018

Duke scientists pinpoint new therapeutic target for macular degeneration

Duke Researchers have pinpointed a new therapeutic target for macular degeneration, an eye disease that affects over 10 million Americans and is the leading cause of blindness in adults over 60.

6 Mar 2018

Gene-editing technology enhances T cell immunotherapies for treating cancer

Last year, the Food and Drug Administration approved the first cellular immunotherapies to treat cancer. These therapies involve collecting a patient's own immune cells -- called T cells -- and supercharging them to home in on and attack specific blood cancers, such as hard-to-treat acute lymphoblastic leukemia and non-Hodgkin lymphoma.

6 Mar 2018

New technique provides pairs of genetically matched cells to study disease

Researchers led by Dr. Knut Woltjen report a new gene editing method that can modify a single DNA base in the human genome with absolute precision.

6 Mar 2018

New and improved CRISPR

David Liu, professor of chemistry and chemical biology at Harvard University is a pioneer of CRISPR-Cas9 gene-editing technology. He has now come up with two new upgrades for this gene editing tool that makes it better than before. These two tools are “cellular detective” and “sharp scissors”.

5 Mar 2018

CRISPR enzyme can also cleave RNA, finds study

Researchers have discovered that the Cas-9 enzyme used in CRISPR gene editing can also cleave complimentary messenger RNA, in addition to DNA.

2 Mar 2018

CRISPR-like gene editing could be potential way to treat patients with harmful blood lipid levels

Using a variation of CRISPR gene editing may be a potential strategy for mimicking the protective effects of a genetic mutation linked to lower cholesterol levels and heart disease risks, according to new mouse research from the Perelman School of Medicine at the University of Pennsylvania published this week in Circulation.

28 Feb 2018

Artificial intelligence methods used for developing precision cancer medicine

A patient's own molecular data can be used to identify, with the use of artificial intelligence methods, the best combinatorial multi-drug therapy for that patient. Network modelling plays a major role in this line of work.

27 Feb 2018

Lab-grown cerebellar cells may help explain how ASD develops at molecular level

Increasing evidence has linked autism spectrum disorder with dysfunction of the brain's cerebellum, but the details have been unclear. In a new study, researchers at Boston Children's Hospital used stem cell technology to create cerebellar cells known as Purkinje cells from patients with tuberous sclerosis complex, a genetic syndrome that often includes ASD-like features.

19 Feb 2018

Gene editing tool used to detect cancer

Researchers have developed a DNA detection system that can detect the presence of bacterial and viral DNA, which could be a valuable diagnostic tool for a range of disorders.

16 Feb 2018

Pioneering research could provide novel insight into how genomic information is read

Pioneering new research could offer a fascinating new insight into how genomic information is read. The human genome contains thousands of genes, but to get the information from a gene requires it to be copied.

13 Feb 2018

Scientists use novel epigenetic-centered techniques to inhibit cancer metastases

In one of the first successes of its kind, researchers from Case Western Reserve University School of Medicine and six other institutions have inhibited the spreading of cancer cells from one part of the body to another.

12 Feb 2018

Scientists develop ultrasound-propelled nanomotors for active delivery of Cas9-sgRNA complex

In cancer research, the “Cas-9–sgRNA” complex is an effective genomic editing tool, but its delivery across the cell membrane to the target (tumor) genome has not yet been satisfactorily solved. American and Danish scientists have now developed an active nanomotor for the efficient transport, delivery, and release of this gene scissoring system.

9 Feb 2018

USC scientists uncover cellular mechanism responsible for ALS and frontotemporal dementia

Scientists have for the first time discovered a mechanism that limits the number of "cellular janitors" in the nervous system, leading to increased risk for two neurodegenerative diseases: amyotrophic lateral sclerosis and frontotemporal dementia, according to a Keck School of Medicine of USC study published today in Nature Medicine.

7 Feb 2018

New CRISPR gene-editing technique can correct majority of mutations that cause DMD

Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.

6 Feb 2018

Researchers report technical advances in production of HIV vaccines

Research on HIV over the past decade has led to many promising ideas for vaccines to prevent infection by the AIDS virus, but very few candidate vaccines have been tested in clinical trials.

6 Feb 2018

Novel method can edit genes with substantially fewer errors

Gene therapy is an emerging strategy to treat diseases caused by genetic abnormalities. One form of gene therapy involves the direct repair of a defective gene, using genome-editing technology such as CRISPR-Cas9. Despite its therapeutic potential, genome editing can also introduce unwanted and potentially harmful genetic errors that limit its clinical feasibility.

5 Feb 2018

The effects of Huntington’s disease can be seen as early as conception, finds new study

In a breakthrough study, scientists at the Rockefeller University have observed the effects of Huntington’s disease in neurons as early as conception.

30 Jan 2018

Gene Editing News and Research

UCLA-developed nanospears could make gene therapies safer, faster and more cost-effective

Researchers uncover link between Parkinson’s disease mutations and plaque formation in the brain

NIH-funded researchers use CRISPR to rapidly find new genetic suspects behind ALS, FTD

Duke scientists pinpoint new therapeutic target for macular degeneration

Gene-editing technology enhances T cell immunotherapies for treating cancer

New technique provides pairs of genetically matched cells to study disease

New and improved CRISPR

CRISPR enzyme can also cleave RNA, finds study

CRISPR-like gene editing could be potential way to treat patients with harmful blood lipid levels

Artificial intelligence methods used for developing precision cancer medicine

Lab-grown cerebellar cells may help explain how ASD develops at molecular level

Gene editing tool used to detect cancer

Pioneering research could provide novel insight into how genomic information is read

Scientists use novel epigenetic-centered techniques to inhibit cancer metastases

Scientists develop ultrasound-propelled nanomotors for active delivery of Cas9-sgRNA complex

USC scientists uncover cellular mechanism responsible for ALS and frontotemporal dementia

New CRISPR gene-editing technique can correct majority of mutations that cause DMD

Researchers report technical advances in production of HIV vaccines

Novel method can edit genes with substantially fewer errors

The effects of Huntington’s disease can be seen as early as conception, finds new study

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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