Help for patients with sickle cell disease may soon come from gene editing to fix the mutation that causes the disease and boost the patient’s own protective fetal hemoglobin. New research shows that using CRISPR-Cas9 and a corrective short DNA template to repair the sickle cell mutation in a patient’s hematopoietic stem cells (HSCs) could…
Natural Gene Therapy for Intractable Skin Disease Discovered
Pathogenic gene mutations causing a type of intractable skin disease can be eliminated from some parts of patients’ skin as they age, according to Hokkaido University researchers and their collaborators in Japan. This represents a form of natural gene therapy. In general, there is no fundamental treatment method capable of curing diseases caused by gene…
Estimating the Efficacy and Cost of Curative Gene Therapy for Beta-Thalassemia
Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem cell transplantation (HSCT). The study, which analyzes and compares the effectiveness and cost of gene therapy versus (HSCT) in patients…
Using CRISPR, Researchers Discover Treatment for Box Jellyfish Sting
Gene-Editing Technology May Produce Resistant Virus in Cassava Plant
New ‘Hairpin’ Approach Takes CRISPR to Next-Level Accuracy
Researchers may have found a way to significantly improve CRISPR/Cas9 gene editing technology. Biomedical engineers from Duke University have added a short tail to the guide RNA that folds back and binds onto itself to create a lock that can only be undone by the targeted DNA sequence, ultimately improving CRISPR’s accuracy by an average…
Russian Scientists Alter 3D Genome Using ‘Small Molecules’
Researchers have discovered that the spatial organization of the genome can be altered using small molecule compounds which are considered as promising anti-cancer drugs. This work opens up the prospect of developing a new class of anticancer epigenetic drugs that alter the 3D genome. The results were published in Nature Communications. The properties of a living…
CRISPR, Transistor Combo Rapidly Detects Genetic Mutations
A new handheld device that combines CRISPR technology with graphene-based electronic transistors can rapidly detect specific genetic mutations. Researchers from the University of California, Berkeley and the Keck Graduate Institute of The Claremont Colleges have created the CRISPR-Chip device that can in just a few minutes diagnose genetic diseases or evaluate the accuracy of other…
Research Suggests New Drug Targets for Osteoporosis
For Infection-fighting Cells, a Guideline for Expanding the Troops
Gene Identified That Increases Risk of Antibiotic Reaction
Researchers at Vanderbilt University Medical Center and colleagues have identified a gene that increases the risk for a severe and potentially life-threatening reaction to the commonly prescribed antibiotic vancomycin. Routine testing for this gene could improve patient safety and reduce unnecessary avoidance of other antibiotics, they reported in the Journal of Allergy and Clinical Immunology.…
New Strategy Improves Efficiency of CRISPR-Cas9 Genome Editing
Genomics Researchers Say ‘Junk’ DNA Key to Advancing Medicine
New Protein Could Deliver Next Gene-Editing Tool
While CRISPR Cas9 remains a mainstay in the genomics world, researchers have now found a much smaller protein called CasX that could be used for gene editing. In 2017, a research team from the University of California Berkeley discovered in some of the world’s smallest bacteria a protein similar to Cas9 but substantially smaller, which…
In Prenatal Testing, ‘Genomics’ Sometimes Sees What Genetic Tests Can’t
New Mutations Causing Inherited Deaf-Blindness Have Been Discovered
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…