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UrduApproach rapidly generates the functional diversity needed to evade neutralization by the immune system, allowing more patients to benefit from gene therapies
Tag: Gene Therapy
Machine-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
Neurons 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 characterized by reduced brain growth after birth, intellectual disability, epilepsy and difficulties with balance and speech. “One of the major…
A 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
Winner-takes-all synthetic gene circuit process opens new pathways to disease treatment
Division of synthetic gene circuit workloads will make therapy more effective.
Registration is open for ISSCR 2021 Virtual Annual Meeting June 21-26
Exceptional science, Enhanced digital experience, Flexible viewing hours
Genethon 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 the lyosomes, which then causes cell damage in various tissues, in particular the heart, the muscles, the liver and the…
February 2021 Issue of Neurosurgical Focus: “Gene and Viral Therapy for Glioblastoma Multiforme”
Announcement of articles in the February issue of neurosurgical Focus.
A 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
Researchers use patients’ cells to test gene therapy for rare eye disease
Scientists at the National Eye Institute (NEI) have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood. A form of Leber congenital amaurosis, the disease is caused by autosomal-dominant mutations in the CRX gene, which are challenging to treat with gene therapy.
First hybrid gene therapy shows early promise in treating long QT syndrome
In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid gene therapy in long QT syndrome, a potentially lethal heart rhythm condition.
Hybrid closed-loop insulin therapy improves glycemic control
New Rochelle, NY, January 26, 2021–Hybrid closed-loop insulin therapy improved glycemic control in adolescents and young adults with type 1 diabetes. These outcomes, derived from the International Diabetes Closed-Loop (iDCL) Trial, are reported in the peer-reviewed journal Diabetes Technology &…
Genome editing to treat human retinal degeneration
New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Technological advances in gene editing, continuing safety concerns, and strategies to overcome these challenges…
Biodistribution of AAV gene transfer vectors in nonhuman primate
New Rochelle, NY, January 15, 2021–The biodistribution of adeno-associated virus (AAV) gene transfer vectors can be measured in nonhuman primates using a new method. The method quantifies whole-body and organ-specific AAV capsids from 1 to 72 hours after administration. Study…
CHOP and Penn Researchers Identify Nanoparticles that Could Be Used in Therapeutic mRNA Delivery before Birth
Researchers at Children’s Hospital of Philadelphia and the School of Engineering and Applied Science at the University of Pennsylvania have identified ionizable lipid nanoparticles that could be used to deliver mRNA as part of fetal therapy. The proof-of-concept study, published today in Science Advances, engineered and screened a number of lipid nanoparticle formulations for targeting mouse fetal organs and has laid the groundwork for testing potential therapies to treat genetic diseases before birth.
Researchers identify nanoparticles that could deliver therapeutic mRNA before birth
Study demonstrates the efficacy and therapeutic potential of select ionizable lipid nanoparticles for delivering mRNA to treat genetic diseases before birth
A CNIO study links severe COVID-19 disease to short telomeres
The study was carried out with data from 89 patients admitted to the IFEMA Hospital
Gene therapy strategy found effective in mouse model of hereditary disease TSC
Treatment reverses aspects of tuberous sclerosis complex, which is characterized by noncancerous tumors in multiple organs.
New clues to prostate cancer
Why aggressive subtype can resist treatment
Brain cancer linked to tissue healing
Brain tumours might arise when tissue does not heal properly– a finding that opens up new ideas about how cancer develops and how to combat it
Scientists at Tel Aviv University develop new gene therapy for deafness
Breakthrough may help in the treatment of children with hearing loss
Salk Assistant Professor Dmitry Lyumkis receives CAREER award from NSF
LA JOLLA–(December 22, 2020) Assistant Professor Dmitry Lyumkis has received a Faculty Early Career Development Program (CAREER) award from the National Science Foundation (NSF). The CAREER award supports early career scientists who serve as academic role models and lead scientific…
Researchers identify a rare genetic bone disorder through massive sequencing methods
They have used precision medicine to uncover and treat new skeletal disorders
Gene therapy for placental insufficiency moves toward the clinic
New Rochelle, NY, December 15, 2020–A new study identified an adenovirus gene therapy vector carrying a VEGF isoform. It can improve uterine blood flow in placental insufficiency, as reported in the peer-reviewed journal Human Gene Therapy . Click here to…
Penn vision researchers honored by End Blindness 2020
The Outstanding Achievement Prize highlights the contributions of the School of Veterinary Medicine’s Gustavo D. Aguirre and the Perelman School of Medicine’s Jean Bennett and Albert M. Maguire toward a gene therapy for a form of blindness.
Exploring how prostate cancer cells resist treatment
Research by a University of Georgia scientist sheds light on how two genes factor into prostate cancer cells becoming resistant to treatment, providing a potential new target for therapeutics.
Gene discovery could help prevent heart attacks
Researchers at the University of Virginia have shed light on how our genes affect our risk for coronary artery disease, the most common form of heart disease. In addition to identifying gene variants that influence risk, they found that one…
Single-eye gene therapy improves vision in both eyes of patients with inherited eye disorder
A gene therapy for an inherited eye disorder can ameliorate vision loss in both eyes despite only being injected into one, according to a phase 3 clinical trial involving 37 patients. The treated patients showed sustainable improvements in vision after…
Gene therapy injection in one eye surprises scientists by improving vision in both
Injecting a gene therapy vector into one eye of someone suffering from LHON, the most common cause of mitochondrial blindness, significantly improves vision in both eyes, scientists have found. In a landmark phase 3 clinical trial, the international team, coordinated…
New Cedars-Sinai Biomanufacturing Center to Spur Cell Therapies
Cedars-Sinai has launched a center to manufacture the next generation of stem cell and gene therapies that will enable biomedical researchers, government medical programs, commercial entities and others to develop new biologic drugs and propel novel disease discoveries.
‘Off switch’ during error-prone cell cycle phase may fix CRISPR’s unwanted changes problem
Turning off gene-editing until it reaches cell cycle phases where more accurate repairs are likely to happen offers a promising fix to CRISPR-Cas9’s problem with unwanted genetic changes
Mimicking the effect of exercise with gene therapy
Hope for people whose illness or injuries mean they can’t exercise
Genetically engineered T cells could lead to therapies for autoimmune diseases
A new study has found that a novel T cell genetically engineered by University of Arizona Health Sciences researchers is able to target and attack pathogenic T cells that cause Type 1 diabetes, which could lead to new immunotherapy treatments.…
Genethon: green light from the ANSM to start a gene therapy trial for Duchenne.D
Genethon received this Monday 30th of November the authorisation from the ANSM to start in France a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004
Retinal transplant boost opens door to treat eyesight loss
Modifying stem cells ahead of retinal transplant can boost chances of success
A new spin-off company to develop the first treatment against pulmonary fibrosis based on telomerase
After several years of research and many joint scientific publications, molecular biologists Maria Blasco (National Cancer Research Centre, CNIO) and Fàtima Bosch (Universitat Autònoma de Barcelona, UAB) have developed a telomerase gene therapy for the treatment of different pathologies related…
IU researchers publish first article dedicated to Hoosier youth’s donated tumor
Tyler Trent, college student and football superfan, captured national spotlight during courageous battle against cancer
Gene therapy gives man with sickle cell disease the chance for a better future
In July 2019, Evie Junior enrolled in a clinical trial for an experimental stem cell gene therapy for sickle cell disease. The study is led by UCLA Broad Stem Cell Research Center physician-scientists Dr. Donald Kohn and Dr. Gary Schiller and funded by the California Institute for Regenerative Medicine.
Researchers tackle sickle cell disease with CRISPR-based gene editing
$1 million grant will advance personalized medicine using gene editing at ChristianaCare’s Gene Editing Institute
New €25.5 million European project to accelerate development of ATMPs
The field of Advanced Therapy Medicinal Products research, which includes gene and cell therapies, is expected to grow exponentially in the coming years, with potentially up to 10-20 new drug applications submitted per year to the FDA by 2025 The…
New findings speed progress towards affordable gene therapy
Researchers used metal-organic frameworks (MOFs) enhanced with a green tea phytochemical coating to target human prostate cancer cells for the first time. The new method for deploying the genetic snipping tool directly into target cells is a big step towards…
Study of ‘exceptional responders’ yields clues to cancer and potential treatments
In a comprehensive analysis of patients with cancer who had exceptional responses to therapy, researchers have identified molecular changes in the patients’ tumors that may explain some of the exceptional responses. The results demonstrate that genomic characterizations of cancer can…
Non-hereditary mutation acts as natural gene therapy in patient with rare disease
Scientists at a research center supported by FAPESP identified a non-inherited mutation in blood cells from a patient with GATA2 deficiency that may have prevented bone marrow failure and other clinical manifestations.
EBMT 47th Annual Meeting
The EBMT is honoured to invite you to the 47th EBMT Annual Meeting, which will take place virtually, 14th-17th March, 2021. More than 6,000 delegates attend the EBMT Annual Meeting. Our top-notch scientific programme for physicians, nurses, data and quality…
Reversal of glial scar tissue back to neuronal tissue through neuroregenerative gene therapy
Brain or spinal cord injury often results in glial scar tissue that is correlated to neural functional loss. Glial scar is a well-known obstacle for neural regeneration due to its dense glial cell composition and lack of functional neurons. A…
First Non-human primate study showing promise of gene therapy for stroke repair
Stroke is a leading cause of death and severe long-term disability with limited treatment available. A research team led by Prof. Gong Chen at Jinan University, Guangzhou, China recently reported the first non-human primate study demonstrating successful in vivo neural…
Risk of AAV mobilization in gene therapy
New Rochelle, NY, November 11, 2020–New data highlight safety concerns for the replication of recombinant adeno-associated viral (rAAV) vectors commonly used in gene therapy. These findings, which emphasize the need for mobilization resistant AAV vectors, are reported in the peer-reviewed…
San Diego zoo global biobanking advances wildlife conservation and human medicine worldwide
In a study that has unprecedented implications to advance both medicine and biodiversity conservation, researchers have sequenced 131 new placental mammal genomes, bringing the worldwide total to more than 250
Penn researchers develop approach to prevent toxicity tied to neurological gene therapy
Advancement improves safety for gene therapies targeting the central nervous system
Induced liver regeneration enhances CRISPR/Cas9-mediated gene repair
New Rochelle, NY, November 9, 2020–Use of thyroid hormone to boost hepatocyte proliferation enhanced the efficiency of CRISPR/Cas9-mediated gene correction in the mouse liver. This dietary induction of hepatocyte regeneration may be a viable clinical strategy to enhance gene repair…