Alliance for Cancer Gene Therapy Summit 2021 features world renowned cancer researchers advancing so
James P. Allison, PhD, Carl H. June, MD and Siddhartha Mukherjee, MD Keynote April 29 Summit
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CNIO scientists discovers a combination therapy for aggressive brain tumors
Glioblastomas are the most common and most aggressive brain tumours. Their survival rate has barely increased over the last 50
Read moreCarnegie Mellon/Yale PNA-based technique an essential part of the gene editing toolkit
In an article published in the April 8 issue of Nature , the National Institutes of Health’s Somatic Cell Gene
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Researchers devise more efficient, enduring CAR gene therapy to combat HIV
A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule.
Read moreBringing gene editing into high schools
Toolkit easily incorporates into any laboratory science course
Read moreTadpole nerve regeneration capacity provides clue to treating spinal cord injury
Nagoya University researchers have identified a gene that plays a crucial role in regenerating neurons of African clawed frog tadpoles,
Read moreGene therapy research shows potential for repairing damage caused by glaucoma and dementia
Scientists at the University of Cambridge have shown in animal studies that gene therapy may help repair some of the
Read morePrime editing enables precise gene editing without collateral damage
The latest gene editing technology, prime editing, expands the “genetic toolbox” for more precisely creating disease models and correcting genetic
Read moreThe third generation of siRNA delivery system makes RNAi therapy feasible
In a new study published in the Cell Research , Chen-Yu Zhang’s group at Nanjing University reports “In vivo self-assembled
Read moreJohn D. Carpten elected fellow of AACR Academy
American Association for Cancer Research honors distinguished USC Norris Comprehensive Cancer Center scientist
Read moreSMART develops rapid deterministic lateral displacement assay to assess immune response
Novel DLD assay is capable of rapidly assessing host inflammatory response, allowing patients exhibiting a life-threatening hyper-aggressive immune response to be identified and treated expeditiously
Read moreResearchers improve plant prime editing efficiency with optimized pegRNA designs
Precision genome editing enables the precise modification of DNA in living cells, thus enabling a breadth of opportunities for plant
Read moreNew anti-cancer therapy: Converting glioma cells into neurons
Glioma is a fatal neurological disorder that has limited interventional treatment, despite extensive research over the past several decades. A
Read moreGene therapy using ‘zinc fingers’ may help treat Alzheimer’s disease, animal study shows
Treatment involving a single injection has long-lasting effects
Read moreAlliance for Cancer Gene Therapy announces Summit 2021 be there when the next breakthroughs happens
Join world renowned cancer researchers as they gather to discuss latest advances in cancer cell and gene therapy for solid tumors
Read moreDavid Schaffer Harnesses “Directed Evolution” for Gene Therapy
Hertz Fellow David Schaffer uses high throughput genetic sequencing technology to identify gene variants that can potentially help restore sight, repair hearts damaged by Fabry disease, and improve lung function in patients with cystic fibrosis.
Read moreScientists create model of an early human embryo from skin cells
For first time, fibroblast-derived model of early embryo will allow extensive study into causes of very early miscarrriage and effects of toxins and drugs on early development
Read moreCellular benefits of gene therapy seen decades after treatment
An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has
Read moreViruses adapt to ‘language of human cells’ to hijack protein synthesis
Findings may boost design of antiviral treatments, gene therapies and vaccines
Read moreSingapore scientists develop novel gene editor to correct disease-causing mutations
The CRISPR-based gene editor, C-to-G Base Editor (CGBE), opens up treatment avenues for up to 40 per cent of genetic disorders caused by single-nucleotide mutations
Read moreWith gene therapy, scientists develop opioid-free solution for chronic pain
A gene therapy for chronic pain could offer a safer, non-addictive alternative to opioids. Researchers at the University of California San Diego developed the new therapy, which works by temporarily repressing a gene involved in sensing pain. It increased pain tolerance in mice, lowered their sensitivity to pain and provided months of pain relief without causing numbness.
Read moreNew spherical nucleic acid ‘drug’ kills tumor cells in humans with glioblastoma
Early clinical trial shows experimental drug crosses blood-brain barrier to trigger death of brain tumor cells
Read moreA new co-driver in breast cancer
UC researchers discover how two genes cooperate to cause cancer growth and can target treatments to stop it from happening
Read moreProtein discovery could help enable eco-friendly fungicides
Findings explain how plants use RNA to defend against fungal invaders
Read moreFunding the future of cancer research
PICI awards nearly $3 million to six early career researchers
Read moreACGT awards grant to advance strategies for treating lung cancer
Recipient Mount Sinai approaching a potential breakthrough in the fight against solid tumors
Read moreCell culture engineering webinars
The in-person Cell Culture Engineering XVII Conference has been postponed until October 16 – October 22, 2021 in Tucson, Arizona.
Read moreOverlooked Cilium Could Be Genetic Key to Common Diseases
Long thought a vestigial part of human cells, new genetic analysis of the primary cilium shows that it may be tied to common conditions like diabetes and kidney failure
Read moreA Canadian success story: world-first to treat Fabry disease with gene therapy
Results of a world-first Canadian pilot study on patients treated with gene therapy for Fabry disease show that the treatment is working and safe.
Read moreJDRF, Lupus Research Alliance and National Multiple Sclerosis Society join forces to decode common m
First joint common mechanisms grants announced
Read moreNew technology shows potential to improve potency and durability benefits in gene therapy
WATERTOWN, Mass. – Gene therapy has traditionally been conceptualized as a one-time, curative treatment option; however, research shows that there
Read moreBringing the Latest Glaucoma Science Directly to the Patient
Four brief, illuminating talks by experts on gene therapy, optic nerve regeneration, the role of artificial intelligence, and the importance of trust between physician and patient.
Read moreScientists use DNA origami to monitor CRISPR gene targeting
CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me
Read moreNew therapeutic target for Huntington’s treatment
New perspective on the role of sRNAs in the disease
Read moreExperts on genomics convene for virtual Mutational Scanning Symposium April 5 – 7
‘Exploring one of the biggest challenges facing genomics today – understanding genetic variants’
Read moreNew therapeutic approach may help treat age-related macular degeneration effectively
Inhibiting a gene involved in the abnormal growth of blood vessels in certain ocular disorders may reduce retinal neovascularization, researchers report in The American Journal of Pathology
Read moreNew drug molecules hold promise for treating fatal child disease
Scientists have identified a way to “rescue” muscle cells that have genetically mutated, paving the way to a possible new
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First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimer’s Disease
UC San Diego researchers have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer’s disease or Mild Cognitive Impairment, a condition that often precedes full-blown dementia.
Read moreFirst-in-human clinical trial to assess gene therapy for Alzheimer’s disease
UC San Diego researchers will inject harmless virus carrying a restorative gene into participants’ brains, where earlier animal studies suggest it may slow, prevent or reverse progression of the neurological disorder
Read moreIU researchers find disease-related gene changes in kidney tissue
Discovery signifies potential for revolutionary new genetic approach to treatment of kidney disease
Read moreMachine-learning how to create better AAV gene delivery vehicles
Machine learning study initiated at the Wyss Institute in collaboration with Google Research enables unprecedented AAV capsid diversification with potential for improving gene therapies
Read moreSTING activation reduces graft-versus-host disease in a mouse model
MUSC Hollings Cancer Center researcher finds loss of STING in recipient dendritic cells increases GVHD severity
Read moreArtificial intelligence generates new diversity of AAV capsids for broader gene therapy
Approach rapidly generates the functional diversity needed to evade neutralization by the immune system, allowing more patients to benefit from gene therapies
Read moreNeurons from blood cells enable researchers to test treatments for genetic brain disease
PROVIDENCE, R.I. [Brown University] — New research provides insights into the treatment of Christianson syndrome (CS), an X-linked genetic disease
Read moreA new vision for AAV-delivered gene therapies
A broadly applicable AAV genome-coupled immunomodulation strategy helps cloak the AAV virus from unwanted immune responses, and offers important insights into ocular inflammation
Read moreWinner-takes-all synthetic gene circuit process opens new pathways to disease treatment
Division of synthetic gene circuit workloads will make therapy more effective.
Read moreRegistration is open for ISSCR 2021 Virtual Annual Meeting June 21-26
Exceptional science, Enhanced digital experience, Flexible viewing hours
Read moreGenethon is delighted about the launch of a gene therapy clinical trial for late-onset Pompe disease
Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in
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February 2021 Issue of Neurosurgical Focus: “Gene and Viral Therapy for Glioblastoma Multiforme”
Announcement of articles in the February issue of neurosurgical Focus.
Read moreA potentially safer, more effective gene therapy vector for blood disorders
New preclinical study shows vector results in significantly more hemoglobin production than vectors currently used in gene therapy for sickle cell disease and beta-thalassemia
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