Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Researchers at the University of Birmingham have discovered a protein fragment called ?N-JARID2 that regulates skin cell differentiation. The finding holds promise for developing new gene therapies for psoriasis and other skin conditions caused by hyper-proliferation of skin cells.
A research group at FAU Erlangen-Nürnberg is developing procedures for monitoring the newly-developed therapies for neuroretinal degeneration. They are using a highly accurate method to measure perception thresholds, which can also be used to track disease progression and test new treatments.
Researchers at UCSF developed CRISPRa, a modified version of the gene-editing tool that activates appetite-suppressing genes without making cuts to the genome. This approach prevented severe obesity in genetically engineered mice with only one functional copy of certain genes.
Researchers at Aarhus University have made a breakthrough in understanding hereditary angioedema by identifying cellular defects leading to the rare disease. The study reveals that genetic mutations cause intracellular retention of C1-inhibitor, resulting in patients producing only 10-20% of normal levels.
A large-scale genetic study has identified 11 new genes associated with epilepsy, which may inform the development of new treatments. The research, led by RCSI researchers, compared DNA samples from over 15,000 people with and without epilepsy, tripling the number of known genetic associations.
Children's Hospital of Philadelphia celebrates European Commission approval of LUXTURNA, a one-time gene therapy treating vision loss due to inherited retinal dystrophy. The therapy has received regulatory approval in both the US and EU, offering new hope for patients with progressive blindness.
In 2018, the biotech industry saw significant investment from venture capital firms and a surge in initial public offerings. Promising developments include the approval of the first RNA interference drug, Onpattro, for treating rare genetic disorders, as well as advancements in CRISPR technology and cannabis-based therapeutics.
The precision medicine initiative is shifting healthcare from population-based approaches to individualized care focusing on each patient's genetic makeup. This shift raises new legal, policy, and ethical issues, including liability, trust, governance, and data access and quality.
Researchers developed niosomes, a type of non-viral vector, to transfer genetic material into the brain. The innovative formulation uses lycopene as a helper lipid, improving transfection efficiency in neuronal and glial cells.
Researchers at the University of Delaware have developed a novel approach to gene therapy using microparticles that deliver gene-regulating material to hematopoietic stem and progenitor cells. This technology could potentially treat inherited blood disorders such as sickle cell anemia and thalassemia by altering the genetic defect in t...
Researchers have developed a gene therapy treatment that is reversing disease symptoms in two adults with Sickle Cell Anemia. The treatment, which uses reduced-intensity preconditioning, shows promise for transportability to resource-challenged parts of the world where SCA is most common.
Scientists have discovered that an emerging Parkinson's gene therapy called AAV2-GAD creates new brain circuits associated with improved motor movement. The therapy has shown therapeutic effects for patients by forming new neural pathways in the brain, connecting the subthalamic nucleus to other motor regions.
Researchers identified a set of genetic variants associated with poorer stroke recovery in patients. The study, involving over 2,000 patients, found that certain variants of the PATJ gene predispose individuals to worse outcomes after a stroke, resulting in significant disability and dependence.
A newly published mouse model of Facioscapulohumeral muscular dystrophy (FSHD) has been developed to test potential therapeutics. The TIC-DUX4 mice express DUX4 only when exposed to the drug Tamoxifen, allowing researchers to control gene expression and study its effects on muscle weakness.
A study published in Human Gene Therapy found that urocortin 3 gene therapy significantly improved left ventricular function and ejection fractions in mice with induced heart failure. The therapy resulted in a 1.9-fold increase in left ventricular function and a 32% relative increase in ejection fractions.
Researchers developed a combinatorial gene therapy approach that inhibits inflammation and promotes pro-anabolic cartilage genes, preserving articular cartilage better than each approach alone. This strategy was shown to be effective in both moderate and severe post-traumatic osteoarthritis models.
Researchers have discovered a gene signature biomarker that can predict which patients will respond to immune therapy based on the extracellular matrix's stiffness around cancer cells. The study suggests that stiffening of the ECM barrier may physically block the immune system.
A genetic study led by University of Helsinki researchers has uncovered the underlying causes of severe heart muscle diseases in children, revealing a unique genetic profile for each family. The study's findings have significant implications for predicting disease course and treatment decisions, paving the way for personalized medicine.
A machine-learning algorithm, inDelphi, predicts the precise correction of broken genes by analyzing data from CRISPR-induced breaks. Researchers successfully corrected nearly 200 disease-associated genetic variants, restoring gene function to healthy states.
New research presents promising results from preclinical studies using gene therapy to treat amyotrophic lateral sclerosis, Parkinson's disease, and other neurological disorders. Gene therapy has been successfully used to slow disease progression and improve symptoms in mouse models.
Researchers developed an antisense oligonucleotide therapy to halt or reverse SCA7-induced vision loss. Studies in mice and humans show promise for treating the rare condition with fewer side effects than existing medications.
The SQZ Biotechnologies platform has been shown to preserve key biological functions essential for cell therapies, unlike electroporation technology. This study highlights the superiority of SQZ in delivering target-specific immune responses and enabling scalable cell therapy development.
Children's hospitals will use electronic health records and DNA samples to identify genetic markers for severe pediatric growth disorders, enabling early diagnosis and targeted treatments.
A new study by UMass Medical School researchers successfully delivers RNAi-based gene therapy to silence SOD1 protein mutations linked to ALS without adverse effects. The therapy, delivered via a viral vector, achieved silencing of over 90% in some motor neurons, suggesting a safe and potentially one-time treatment for humans.
Researchers have found associations between addiction-related phenotypes and variants in the PTPRD gene, a neuronal cell adhesion molecule. A chemical compound, 7-BIA, has been shown to selectively inhibit phosphatase activity of PTPRD in mice, reducing cocaine self-administration rates.
Researchers found that patients with a non-functional IDO2 gene had more favorable outcomes when receiving radiotherapy, living cancer-free for almost twice as long. The discovery could lead to personalized treatment recommendations and improve patient survival rates.
Researchers describe a DNA immunization platform that stimulates innate and adaptive immunity to produce high levels of antigen-specific antibody responses. The approach activates multiple pathways of innate immunity, leading to the development of high-affinity antibodies in mouse, rabbit, and human models.
Researchers developed a novel DNA vaccine that protected mice against multiple H3N2 viruses and severe illness, offering a promising approach to elicit an immune response against diverse strains. The vaccine strategy represents a step forward in eliminating the need for seasonal reformulation of influenza vaccines.
Researchers at the University of Pennsylvania School of Medicine have discovered that viral vectors used in gene therapy undergo spontaneous changes during manufacturing, affecting their structure and function. The team has developed new ways to prevent these changes, leading to more efficient and safer delivery of gene therapies.
Warren Ruder, a Pitt engineer, has been awarded $1.5 million from the NIH Director's New Innovator Award to develop magnetically induced synthetic gene networks for cell and tissue therapies. His research combines biology and engineering to create new biomimetic systems that can regulate disease pathways.
A study comparing DNA and RNA data from Nigerian breast cancer patients to those in a US database found aggressive molecular features prevalent in tumors from Nigerian women, which could explain disparities in breast cancer mortality. The study highlights the potential for targeted therapies to reduce mortality gap.
MIT biological engineers have developed a way to regulate RNA expression, giving precise control over the dose of therapeutic protein a patient receives. This technology allows doctors to tailor treatment for individual patients and offers a quick way to turn off protein production if necessary.
A multidisciplinary team of scientists from the University of Pennsylvania, Children's Hospital of Philadelphia, and University of Wisconsin-Madison are developing a stem cell-based approach to treat blindness in dogs. The researchers aim to generate specialized retinal cells using adult stem cells and transplant them into the retina.
Researchers have developed a two-pronged approach targeting Ebola virus infection using linked nucleic acid (LNA) antisense oligonucleotides (ASOs). The study demonstrates effective targeting of two Ebola viral genes and the ability to prevent infectivity via Niemann-Pick C1.
Researchers used a novel method to analyze individual brain cells in mice, revealing specific genes that may play roles in traumatic brain injury. Treatments targeting these genes, such as thyroid hormone therapy, showed promise in improving learning and memory tasks.
Researchers at Johns Hopkins Medicine used an endoscopic procedure to deliver therapeutic genes to the liver via the common bile duct, demonstrating a safe and effective method for gene therapy. The technique, called ERCP, was found to be less invasive than traditional injection methods, with no biliary or liver injury observed.
Kathryn Whitehead's project uses genetic engineering to isolate and modify human cells in breast milk for infant disease therapy. The goal is to develop non-invasive treatments for babies with diseases, such as spina bifida or enterocolitis.
Researchers discovered that driver gene mutations are remarkably similar across different metastases from the same patient, offering hope for successful targeted therapies. This finding suggests that single biopsies can capture essential information for therapeutic decision-making.
Researchers developed a genome editing tool to target and eliminate damaged mitochondrial DNA, resulting in improved mitochondrial function in mice. The treatment, known as mtZFN, could provide a practical route to treating patients with mitochondrial diseases.
The RAC's elimination would shift review responsibility to local Institutional Biosafety Committees. The new approach aims to reduce redundancies between FDA and RAC roles in human gene therapy.
A promising gene therapy for visually impaired sheep has been approved for human trials, offering hope for patients with achromatopsia. The treatment has already shown success in treating affected sheep and demonstrates the potential for a cure.
A study led by the University of Exeter Medical School found that disrupting genes and pathways regulating splicing factors can reverse signs of aging in cells. Disrupting ERK and AKT pathways reduced senescent cells, increasing splicing factors and leading to cellular rejuvenation.
Researchers at the Salk Institute and University of Florida used cryo-electron microscopy to image an AAV2 virus with improved resolution. The study reveals key changes in the viral portal that enable it to be less infectious, potentially informing gene therapy applications for various diseases.
A team of researchers has developed a gene therapy cure for the Leber Congenital Amaurosis, a genetic form of childhood blindness. The treatment restores vision to treated children and adults, paving the way for new treatments for genetic conditions.
Researchers at Stanford Medicine have successfully developed a method to induce tolerance to gene therapy in mice, eliminating the autoimmune reaction that often occurs. This breakthrough could lead to effective treatments for single-gene disorders such as Duchenne muscular dystrophy.
Researchers at Gladstone Institutes identify 156 genes affecting ATP levels in cells, shedding light on cellular energy production. The findings could lead to the development of new therapies for diseases such as mitochondrial disorders, heart disease, and neurodegenerative diseases.
Studies with rodent models show reduced anxiety and improved brain function with early treatment using the histone deacetylase inhibitor LBH589. This approach may offer a new therapeutic strategy for Huntington disease.
A recent study has identified 24 genes that are differentially expressed in rats with cerebral aneurysms compared to those without. These genes may enable the development of diagnostic biomarker tests for patients at increased risk of cerebral aneurysm formation.
The NIH grant will support research on biological treatments and clinical diagnosis for patients with hearing loss, a condition affecting over 28 million Americans. Xue Zhong Liu's team will expand a genomic database and assess the impact of genomic testing on patients.
Researchers developed a single gene therapy vector that preserved retinal structure and function for over 8 months in a canine model of autosomal dominant retinitis pigmentosa. The treatment had potential implications for treating inherited retinal degeneration associated with the rhodopsin gene.
Scientists have developed a novel gene therapy that effectively reduces rhodopsin production and prevents photoreceptor death in dogs with autosomal dominant retinitis pigmentosa. The treatment, which combines shRNA interference with a replacement gene, shows promise for slowing or preventing vision loss in humans.
Researchers at CNIO develop gene therapy with telomerase, proving effective in mice against diseases caused by excessive telomere shortening. The study finds that the gene therapy does not increase the risk of developing cancer, even in a cancer-prone setting.
Gene therapy using optimized AAV to deliver human factor VIII showed substantial hFVIII expression and no detectable antibody response in cynomolgus macaques. The results suggest that AAVhu37-based gene therapy has the potential to advance to clinical trials for treating hemophilia A.
Researchers developed a gene therapy that eliminates the abnormal copy of rhodopsin and restores it with a healthy copy, preserving retina's light-sensing photoreceptor cells. This approach has the potential to treat a large percentage of patients with rhodopsin autosomal dominant retinitis pigmentosa.
Scientists have identified nearly 200 potential drug targets for tuberculosis, a complex disease caused by bacteria made up of 4,000 genes. The research found that specific human proteins, such as CBL, can limit bacterial infections and potentially provide a new approach to combat the disease.
A study found significant improvement in cone function and no abnormalities in treated eyes for up to six years. The treatment used an AAV5 vector delivered a CNGA3 gene therapy, offering hope for treating genetic blindness.
A new study shows that a single injection of AAVB1-GAA gene therapy prolonged survival and improved enzyme activity in a mouse model of Pompe disease. The therapy also targeted the respiratory system, improving ventilatory measures.
A new natural history study of Amish nemaline myopathy provides a platform for exploring gene replacement therapy. The study's findings show promise for treating the lethal disorder, which is linked to a mutation of the TNNT1 gene.
A study by University of Utah Health scientists reveals that variation in the genetic modifier Baldspot can influence disease severity in retinitis pigmentosa. The research suggests targeting genes like Baldspot could lead to effective treatments against some disorders.
A new article explores the role of inflammatory cytokines in creating T cell exhaustion in cancer, suggesting a direct pathway for therapeutic interventions. The study found that reprogramming exhausted T cells and anti-inflammation therapy may improve antitumor activity.