A team of scientists from Sechenov First Moscow State Medical University (MSMU) together with their colleagues from leading scientific centers of Moscow and India described a number of genetic mutations causing Usher syndrome (inherited deaf-blindness). They found previously unstudied unique mutations in investigated DNA regions. The results of the study were published in the Ophthalmic Geneticsjournal.…
Whole Genome Sequencing Method May Speed Personalized Treatment of Drug-Resistant Infections
Orchard Therapeutics to Build New Gene Therapy Manufacturing Facility in California
CRISPR Method for Conditional Gene Regulation
A team of engineers at the University of Delaware has developed a method to use CRISPR/Cas9 technology to set off a cascade of activities in cells, a phenomenon known as conditional gene regulation. Their method, described in the journal Nature Chemical Biology, introduces a new functionality to CRISPR, one of today’s most-talked-about technologies. Gene editing…
CRISPR Gene Editing Will Find Applications in Plastic and Reconstructive Surgery
Breakthrough: Promising New Genetic Target for Immunotherapy
Following the 2018 Nobel Prize for Medicine, global attention is now more than ever turned toward the promise of immunotherapy in oncology. An international team’s work has shed new light on a molecule called TIM-3 that might play a key role in the regulation of the immune response. Scientists and physicians from the Research Institute…
Novel Combination Therapy Promotes Wound Healing
By incorporating a gene-suppressing drug into an over-the-counter gel, researchers at Albert Einstein College of Medicine and their colleagues cut healing time by half and significantly improved healing outcomes compared to control treatments. Results from the combination therapy, which was tested in mice, were published online today in Advances in Wound Care. “Not only did wound…
New CRISPR Tool Opens Up More of the Genome for Editing
The genome editing system CRISPR has become a hugely important tool in medical research, and could ultimately have a significant impact in fields such as agriculture, bioenergy, and food security. The targeting system can travel to different points on the genome, guided by a short sequence of RNA, where a DNA-cutting enzyme known as Cas9 then…
Novel DNA Vaccine Design Offers Broad Protection Against Influenza-A H3N2
Researchers Modify CRISPR to Reorganize Genome
Researchers at Stanford University have reworked CRISPR-Cas9 gene-editing technology to manipulate the genome in three-dimensional space, allowing them to ferry genetic snippets to different locations in a cell’s nucleus. The new technique, dubbed CRISPR-genome organization or simply CRISPR-GO, uses a modified CRISPR protein to reorganize the genome in three dimensions. If CRISPR is like molecular…
A New Way to Manufacture Small Batches of Biopharmaceuticals on Demand
CRISPR Helps Produce Bigger, Healthier Tomatoes
Decoding the Structure of an RNA-Based CRISPR System
Next-Generation Sequencing: The Need for Quality Control
CRISPR Screen Identifies Gene That Helps Cells Resist West Nile, Zika Viruses
Gilead Sciences and Precision BioSciences Collaborate to Develop Therapies Against Hepatitis B Virus Using Genome Editing
Gilead Sciences and Precision BioSciences announced today that the companies have entered into a strategic collaboration to develop therapies targeting the in vivo elimination of hepatitis B virus (HBV) with Precision’s proprietary genome editing platform, ARCUS. An estimated 257 million people are living with HBV infection around the world. Current HBV treatments suppress HBV viral replication but…
Hspgo: More Than Just a Chaperone
Molecular Hopper Can Move Individual DNA Strands
A research team from the University of Oxford has developed a molecular hopper that is small enough to be able to move single strands of DNA through a protein nanotube. The device works by making and breaking in sequence simple chemical bonds that attach it to a nanoscale track that can be turned on, off…
CRISPR Halts Duchenne Muscular Dystrophy Progression in Dogs
Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline. The research published in Science documents unprecedented improvement in the muscle…
Predicting How Splicing Errors Impact Disease Risk
No one knows how many times in a day, or even an hour, the trillions of cells in our body need to make proteins. But we do know that it’s going on all the time, on a massive scale. We also know that every time this happens, an editing process takes place in the cell…
New Method Grows Brain Cells From Stem Cells Quickly and Efficiently
CasPER: A New Method For Diversification of Enzymes
A new study published in the Metabolic Engineering Journal describes a method based on CRISPR/Cas9, which enables flexible engineering of essential and nonessential enzymes without additional engineering. This could be of great importance for various aspects including the development of bio-based production of pharmaceuticals, food additives, fuels and cosmetics. “When having a production strain this will make…
Approval of First ‘RNA Interference’ Drug – Why the Excitement?
Small interfering RNA sounds like something from a science fiction novel rather than a revolutionary type of medicine. But this odd-sounding new drug offers a novel strategy for treating disease by targeting the root cause rather than just the symptoms. This is an exciting approach because it enhances the effectiveness of the treatment and reduces…
