Thailand Hub of Talent for Cancer Immunotherapy International Conference

The Faculty of Medicine, Chulalongkorn University, cordially invites all to attend the “Thailand Hub of Talent for Cancer Immunotherapy International Conference: Portal to Global Collaboration for Next Generation Cell and Gene Therapy Development” on November 16-17, 2023 at Bhumisiri Mangklanusorn Building, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand.

CHOP and Penn Medicine Researchers Develop “In Vivo” RNA-based Gene Editing Model for Blood Disorders

In a step forward in the development of genetic medicines, researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body. If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant. The findings were published today in the journal Science.

New understanding of why kidney cancers become metastatic discovered by MD Anderson researchers

Researchers at The University of Texas MD Anderson Cancer have engineered a new model of aggressive renal cell carcinoma (RCC), highlighting molecular targets and genomic events that trigger chromosomal instability and drive metastatic progression.
The study, published today in Nature Cancer, demonstrates that the loss of a cluster of interferon receptor (IFNR) genes plays a pivotal role in allowing cancer cells to become tolerant of chromosomal instability. This genomic feature may be used to help clinicians predict a tumor’s potential to become metastatic and treatment resistant.

Researchers Demonstrate First Precision Gene Editing in Miscanthus

Miscanthus thrives on marginal lands with limited fertilization and tolerates drought and cool temperatures, making it an ideal bioenergy candidate. Previous efforts to genetically improve miscanthus focused on introducing external genes at random places in the plant’s genomes. This research developed gene-editing procedures using CRISPR/Cas9 that will allow scientists to selectively target existing genes to knock out their function and introduce new genes into precise locations.

Transforming plants into allies in the fight against climate change

Nature-based solutions are an effective tool to combat climate change triggered by rising carbon emissions, whether it’s by clearing the skies with bio-based aviation fuels or boosting natural carbon sinks. At the Department of Energy’s Oak Ridge National Laboratory, scientists are leading research to transform plants into key drivers of decarbonization, from creating biomass crops for new fuels to enhancing the ability of plants to absorb and store carbon.

High School Students Learn the Basics of Base Editing to Cure “GFP-itis”

Genome editing is used to modify the genes of living organisms to elicit certain traits, such as climate-resilient crops or treating human disease at the genetic level. It has become increasingly popular in agriculture, medicine and basic science research over the past decade, and will continue to be relevant and utilized well into the future.

Improved Gene Editing Method Could Power the Next Generation of Cell and Gene Therapies

A new approach to the genetic engineering of cells promises significant improvements in speed, efficiency, and reduction in cellular toxicity compared to current methods. The approach could also power the development of advanced cell therapies for cancers and other diseases, according to a study from researchers in the Perelman School of Medicine at the University of Pennsylvania.

Hijacking the Hijackers: Engineering Bacterial Viruses to Genetically Modify their Hosts

Most methods of editing bacterial genomes use plasmids to transfer DNA between bacterial cells, but this approach isn’t always efficient in mixed microbial communities. This research instead developed a new phage-based DNA delivery tool that leverages these viruses’ ability to inject DNA into host bacteria. The researchers also used this tool to edit individual genes inside a target host organism within a living microbial community.

Experts from 14 Nations Discuss Global Gene Drive Project Registry

UC San Diego Herbert Wertheim School of Public Health and Human Longevity Science led 70 participants from 14 nations in a discussion on the ways in which a gene drive project registry could both contribute to and detract from the fair development, testing and use of gene-drive modified organisms.

New technique makes gene editing at scale possible in animals, turning years of work into days and making new kinds of genetic experiments possible

A new gene editing technique developed by University of Oregon researchers compresses what previously would have been years of work into just a few days, making new kinds of research possible in animal models.

A ‘factory reset’ for the brain cures anxiety, drinking behavior

Gene editing may be a potential treatment for anxiety and alcohol use disorder in adults who were exposed to binge drinking in their adolescence, according to the results of an animal study published in the journal Science Advances. The researchers used a gene-editing tool called CRISPR-dCas9 in their experiments to manipulate the histone acetylation and methylation processes at the Arc gene in models of adult rats.

Genetics/biotech expert offers comments & availability on IVG (in vitro gametogenesis) major breakthrough

A groundbreaking study demonstrating the most advanced form of in vitro gametogenesis (making eggs from stem cells, IVG) was published Thursday in Science. See STAT’s coverage of the study.  Regarding the study and breakthrough, Dr. Kevin Doxzen offers the below comments…

CHOP Researchers Use Base Editing in Preclinical Model to Correct Lethal Lysosomal Storage Disease Before Birth

Adding to the growing body of literature demonstrating the feasibility of correcting lethal genetic diseases before birth, researchers at Children’s Hospital of Philadelphia (CHOP) have used DNA base editing in a prenatal mouse model to correct a lysosomal storage disease known as Hurler syndrome. Using an adenine base editor delivered in an adeno-associated viral vector, the researchers corrected the single base mutation responsible for the condition, which begins before birth and affects multiple organs, with the potential to cause death in childhood if untreated.

Gene-Editing ‘Scissor’ Tool May Also be a ‘Dimmer Switch’

In a series of experiments with laboratory-cultured bacteria, Johns Hopkins scientists have found evidence that there is a second role for the widely used gene-cutting system CRISPR-Cas9 — as a genetic dimmer switch for CRISPR-Cas9 genes. Its role of dialing down or dimming CRISPR-Cas9 activity may help scientists develop new ways to genetically engineer cells for research purposes.

Digging Deep For Differences In Duchenne Muscular Dystrophy

DALLAS – Dec. 21, 2020 – A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchene muscular dystrophy (DMD) in humans. The findings, published online recently in PNAS, could lead to new treatments for this devastating degenerative disease and insights into factors that affect muscle development.

Cancer-Fighting Gene Restrains ‘Jumping Genes’

DALLAS – Oct. 29, 2020 – About half of all tumors have mutations of the gene p53, normally responsible for warding off cancer. Now, UT Southwestern scientists have discovered a new role for p53 in its fight against tumors: preventing retrotransposons, or “jumping genes,” from hopping around the human genome. In cells with missing or mutated p53, the team found, retrotransposons move and multiply more than usual. The finding could lead to new ways of detecting or treating cancers with p53 mutations.

Regulators Classify Gene-Edited Rice Varieties with Disease Resistance as Equivalent to Conventional Varieties

The Healthy Crops team, with support from the Bill & Melinda Gates Foundation, have used gene editing tools to develop new varieties of disease-resistant rice that regulators in the United States and Colombia have determined are equivalent to what could be accomplished with conventional breeding.

American Chemical Society’s president comments on award of 2020 Nobel Prize in Chemistry

On behalf of the American Chemical Society (ACS), President Luis Echegoyen, Ph.D., congratulates today’s winners of the Nobel Prize in Chemistry: Emmanuelle Charpentier, Ph.D., and Jennifer A. Doudna, Ph.D. The Royal Swedish Academy of Sciences awarded the prize “for the development of a method for genome editing.”

Danforth Center Scientists Collaborate On $13 Million Bioenergy Project

The U.S. Department of Energy (DOE) awarded a five-year, $13 million grant to a nationwide research project to genetically strengthen Thlaspi arvense, commonly known as pennycress, for use in sustainable energy efforts.

Scientists Aim Gene-Targeting Breakthrough Against COVID-19

Scientists at Berkeley Lab and Stanford have joined forces to aim a gene-targeting, antiviral agent called PAC-MAN against COVID-19.

Grant will help scientists break new ground in gene editing

A new grant from the National Institutes of Health will allow Iowa State University scientists to continue to develop gene editing technologies to model human disease in zebrafish. The research aims to build new tools to determine which genes have therapeutic potential to treat human genetic diseases that affect the cardiovascular, immune and nervous systems.

“CRISPR: A Screener’s Guide” Headlines the March Edition of SLAS Discovery

The March edition of SLAS Discovery features the cover article, “CRISPR: A Screener’s Guide,” by Carlos le Sage, Ph.D., Steffen Lawo, Ph.D., and Benedict C.S. Cross, Ph.D., (Horizon Discovery, United Kingdom). In their review, the authors discuss how CRISPR-Cas9 systems are being used widely throughout the drug discovery process and the development of new precision medicines.

Opening Up DNA to Delete Disease

Protein editorial assistants are clearing the way for cut-and-paste DNA editors, like CRISPR, to access previously inaccessible genes of interest. Opening up these areas of the genetic code is critical to improving CRISPR efficiency and moving toward futuristic, genetic-based assaults on disease. The DNA-binding editorial assistants were devised by a U.S.-based team of bioengineers, who describe their design in APL Bioengineering.