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UrduA new study in mice finds that a gene therapy developed by a UCLA researcher appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements and recurrent seizures.
Tag: Gene Therapy
Scientists Gain Ground on Rare Congenital Neurological Disorder
Two recent discoveries co-led by scientists at Cedars-Sinai may help lead to new ways to treat patients with Allan-Herndon-Dudley syndrome (AHDS), a brain development disorder that causes severe intellectual disability and problems with movement.
Change of temperature causes whole body reprogramming
Human beings, like most organisms, are constantly exposed to alternating colder or warmer temperatures.
Children’s Hospital of Philadelphia Awards Gold Medal to Dr. Katherine High, Gene Therapy Pioneer
In an honor reserved for only the most significant achievements in advancing children’s health, Children’s Hospital of Philadelphia (CHOP) has awarded its Gold Medal to Katherine High, MD, a gene therapy pioneer and one of the lead developers of the first in vivo gene therapy approved by the Food and Drug Administration (FDA). Bestowed only 12 times in CHOP’s 167-year history, the Gold Medal highlights Dr. High’s groundbreaking discoveries at CHOP, which led to a gene therapy treatment for a rare form of inherited blindness and advanced gene therapy for hemophilia to late-stage testing.
Gene Therapy Could Treat Pitt-Hopkins Syndrome, Proof-of-Concept Study Suggests
University of North Carolina School of Medicine scientists, who report their results in the journal eLife, devised an experimental, gene-therapy-like technique to restore the normal activity of the gene deficient in people with Pitt-Hopkins syndrome.
Gene Therapy Reverses Effects of Autism-Linked Mutation in Brain Organoids
UC San Diego scientists use lab-grown human brain tissue to identify neural abnormalities in Pitt-Hopkins Syndrome and show gene therapy tools can rescue neural structure and function.
Cardiologists, Cardiothoracic Surgeons Available for Expert Commentary Ahead of Upcoming Conferences
Cardiologists and cardiothoracic surgeons from the Smidt Heart Institute at Cedars-Sinai are available to discuss the latest advances in research, clinical care, transcatheter procedures and cardiothoracic surgery throughout the International Society for Heart and Lung Transplantation (ISHLT) Scientific Sessions 2022 and Heart Rhythm 2022.
Gene Therapy Shows Early Promise as Angelman Syndrome Treatment
Scientists publish encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities.
UCI receives 5-year, $5 million CIRM award for training of diverse researchers
The University of California, Irvine has received a five-year, $5 million award from the California Institute for Regenerative Medicine to support a comprehensive doctoral, postdoctoral and clinical researcher training program to prepare the current and next generation of leaders in stem cell biology, gene therapy and regenerative medicine.
New Blood: Lab-Grown Stem Cells Bode Well for Transplants, Aging Research
UC San Diego researchers develop a method to grow hematopoietic stem cells in culture, with clinical implications for bone marrow transplants and aging research.
Mirus Bio Introduces the VirusGEN® GMP Product Line for Large-scale Therapeutic Adeno-associated Virus and Lentivirus Production
Mirus Bio, an innovator in transfection technologies, today announces the launch of the TransIT® VirusGEN® GMP product line.
Children’s Hospital of Philadelphia Researchers Develop “Dimmer Switch” to Help Control Gene Therapy
In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a “dimmer switch” system that can control levels of proteins expressed from gene therapy vectors. The system is based on alternative RNA splicing using an orally available small molecule and works effectively in tissues throughout the body, including the brain. The first research regarding this innovation was published today in the journal Nature.
Mount Sinai researchers develop novel therapy that could be effective in many cancers
New York, NY (July 23, 2021) — Mount Sinai researchers have developed a therapeutic agent that shows high effectiveness in vitro at disrupting a biological pathway that helps cancer survive, according to a paper published in Cancer Discovery , a…
The gene’s-eye view of evolution
New book reviews the history of the gene’s-eye view of evolution by describing current disagreements and conceptual debates, and highlighting its value in contemporary evolutionary biology
Study identifies MET amplification as driver for some non-small cell lung cancers
CU Cancer Center researchers get positive results treating patients with MET-inhibitor drug crizotinib
New Evidence Prompts Further Concern for a Key Viral Tool Used in Gene Therapy
Recombinant adeno-associated viruses (rAAV) have long been a key tool used in developing potential gene therapies for a wide range of diseases. But a new study found that rAAV specifically targeted certain brain cells in adult mice, causing the cells…
NIH-funded study finds gene therapy may restore missing enzyme in rare disease
Results provide hope for children with aromatic L-amino acid decarboxylase deficiency
Children’s National Hospital joins the Mendelian Genomics Research Consortium, receiving $12.8 million
The National Institutes of Health’s National Human Genome Research Institute award will help discover inherited rare diseases and conditions in children
Quantum physics helps destroy cancer cells
Researchers have found a way to enhance radiation therapy using novel iodine nanoparticles
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.
Innovative Gene Therapy ‘Reprograms’ Cells To Reverse Neurological Deficiencies
A new method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study was led by researchers at The Ohio State University Wexner Medical Center and The Ohio State University College of Medicine.
Innovative gene therapy ‘reprograms’ cells to reverse neurological deficiencies
A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study, led by researchers at The Ohio State University Wexner Medical Center and The…
Genome editing for food: how do people react?
Research team from the Universities of Göttingen and British Columbia investigates evaluations of breeding technology
Research reveals structure of nanomachine that assembles a cell’s energy control system
Sussex researchers have determined the structure of a tiny multi-protein biological machine, furthering our understanding of human cells and helping to enhance research into cancer, neurodegeneration and other illnesses
Making sense of antisense gene silencing
Researchers from Tokyo Medical and Dental University (TMDU) find proteins that bind to and regulate tocopherol-conjugated heteroduplex oligonucleotides during gene silencing Tokyo, Japan – Gene silencing therapies are used to interfere with, or “silence”, the expression of genes that are…
Three-in-one approach boosts the silencing power of CRISPR
A newly developed CRISPR-Cas9-based tool carries out efficient and long-term gene silencing by epigenetic editing
New research moves novel gene therapy for heart failure closer to the clinic
Research at Baylor College of Medicine, the Texas Heart Institute and collaborating institutions is moving a novel promising gene therapy to treat heart failure closer to the clinic. Published in Science Translational Medicine , the study showed that knocking down…
Preliminary results of clinical trial for Crigler-Najjar syndrome
Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Genethon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of the Liver) annual International Liver Congress on June 26.…
The ISSCR 2021 Annual Meeting starts 21 June
Six days of exceptional stem cell science
LIM domain only 1: One gene, many roles in cancer
Researchers detail another gene target that could be the crucial link to curing different cancers
Scientists uncover the mysteries of how viruses evolve
Scientists uncover the mysteries of how viruses evolve
Advancing manufacture of cell and gene therapies webinar
An ECI webinar on July 21, 2021
New adaptable nanoparticle platform enables enhanced delivery of gene therapies
Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo. The work, led by researchers from RCSI University of Medicine…
Controlling insulin production with a smartwatch
Many modern fitness trackers and smartwatches feature integrated LEDs. The green light emitted, whether continuous or pulsed, penetrates the skin and can be used to measure the wearer’s heart rate during physical activity or while at rest. These watches have…
Study supports gene therapy as a promising treatment for soft bone disease
A single dose of AAV8-TNAP-D10 may be safe and effective for hypophosphatasia
Genetic base editing treats sickle cell disease in mice
Converting a pathogenic hemoglobin gene to a benign variant enables healthy blood cell production in an animal model of sickle cell disease
Elevating African cichlid fish as a scientific model of social disorders
University of Houston researcher wins prestigious Beckman Young Investigator Award
Scientists discover a new genetic form of ALS in children
NIH- and USU- led study links ALS to a fat manufacturing gene and maps out a genetic therapy
The ISSCR releases updated guidelines for stem cell research and clinical translation
Skokie, IL – The International Society for Stem Cell Research (ISSCR), today released updated guidelines for stem cell research and its translation to medicine. The update reflects emerging advances including, stem cell-based embryo models, human embryo research, chimeras, organoids, and…
First clinical trial to assess Alzheimer’s gene therapy receives $5 million
The National Institute on Aging (NIA), part of the National Institutes of Health, has awarded a $5 million grant to researchers at University of California San Diego School of Medicine to conduct a first-in-human Phase 1 clinical trial of a…
Predicting chemotherapy response and tailoring treatments for pancreatic cancer patients
Dartmouth researchers are about to bring change to the treatment of pancreatic cancer with a novel biomarker technology that predicts chemotherapy response and potential benefit of epigenetic therapy in patients with pancreatic cancer
Chemical changes to peptide siRNA-carrier enhance gene silencing for future cancer drugs
MUSC Hollings Cancer Center researchers improve peptide siRNA drug carriers for use as a novel cancer treatment
Accurate evaluation of CRISPR genome editing
Researchers from IDC Herzliya and Bar-Ilan University develop a novel software tool to quantify potential CRISPR-induced errors
Researchers closer to gene therapy that would restore hearing for the congenitally deaf
CORVALLIS, Ore. – Researchers at Oregon State University have found a key new piece of the puzzle in the quest to use gene therapy to enable people born deaf to hear. The work centers around a large gene responsible for…
New report indicates annual economic impact of human genetics/genomics
Grew over $200 billion since 2010, outlines new areas of promising application
Cholesterol levels sustainably lowered using base editing
Base editing is a novel gene editing approach that can precisely change individual building blocks in a DNA sequence. By installing such a point mutation in a specific gene, an international research team led by the University of Zurich has…
City of Hope and Griffith University develop direct-acting antiviral to treat COVID-19
An international team of scientists from the Menzies Health Institute Queensland (MHIQ) at Griffith University and from City of Hope, a research and treatment center for cancer, diabetes and other life-threatening diseases in the U.S., have developed an experimental direct-acting…
New Genetic ‘CopyCatchers’ Detect Efficient and Precise CRISPR Editing in a Living Organism
Scientists have developed a novel CRISPR-based genetic sensor called a “CopyCatcher” to detect instances in which a genetic element is copied precisely from one chromosome to another in cells of a fruit fly.
Gene Therapy in Alzheimer’s Disease Mouse Model Preserves Learning and Memory
Researchers at UC San Diego have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer’s disease, a key step toward eventually testing the approach in humans with the neurodegenerative disease.
Penn Medicine and Children’s Hospital of Philadelphia to Host Symposium on the Future of Cell and Gene Therapies
Penn Medicine and Children’s Hospital of Philadelphia (CHOP) will host a virtual event on May 6 and 7 that will bring together cell and gene therapy leaders from the two institutions and around the world to discuss the latest achievements in the field, novel strategies, and future developments and applications for chimeric antigen receptor, CAR, T cell therapy and more.