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 SISSA have developed a gene therapy approach targeting glioblastomas by introducing an active version of the Emx2 gene, which inhibits astrocyte growth and leads to tumour cell suicide. The treatment has demonstrated efficacy in both vitro and in vivo tests, with potential for preventing aggressive recurrence development.
Researchers evaluated an IHC screening tool for ROS1 gene rearrangements in a cohort of 170 patients. The results showed high sensitivity and specificity rates, making it a feasible option for first-line screening in a lung cancer setting.
Researchers develop an experimental therapy that shuts down the Olig2 gene, halting tumor growth and blocking tumor formation. The approach uses a gene therapy to eliminate Olig2-positive cells, sensitizing them to targeted molecular treatment.
Researchers found that genetically modifying oncolytic viruses to express a junction opening (JO) protein significantly enhances their ability to penetrate and kill malignant epithelial cells. This breakthrough could improve cancer therapy for bulkier solid tumors.
Researchers discuss zebrafish as a model system for studying normal hematopoiesis and blood disorders. The review highlights the knowledge gained from studies of zebrafish, including large-scale forward genetic screens and modeling of human genes to create transgenic zebrafish.
Researchers at the University of Missouri have developed a gene replacement therapy to treat and control Spinal Muscular Atrophy with Respiratory Distress Type 1 (SMARD1). The therapy, administered in a single dose, has shown significant improvements in muscle strength, protein expression, and life span in SMARD1 mice. This study provi...
Researchers at UT Southwestern Medical Center discovered that Fanconi anemia genes play a crucial role in selective autophagy, particularly in removing damaged mitochondria. This finding has implications for treating Fanconi anemia and certain cancers, including familial breast and ovarian cancer.
Scientists used gene therapy to halt pulmonary hypertension progression in a large animal model. The approach delivered SERCA2a gene via aerosol inhalation, slowing vascular changes and improving heart function. Further studies are needed to assess long-term efficacy and safety before human clinical trials.
A pioneering gene therapy has restored vision to patients with a rare form of genetic blindness for up to four years, providing sustained improvement and raising hopes for a cure. The treatment involves injecting a virus into the eye to deliver billions of healthy genes, replacing a key missing gene.
Researchers developed a method demonstrating that independent DNA variants linked to a disease share similar biological properties. This discovery provides a more comprehensive picture of disease biology and offers the opportunity to broaden our understanding of disease and identify new therapeutic targets.
Researchers at Lund University have found a promising new therapeutic target to treat bacterial infections, regulating the immune response and avoiding destructive inflammation. The discovery of IRF-7 as an immunotherapeutic target holds promise for non-antibiotic therapy.
A new study reveals that a genetic risk factor for five major psychiatric diseases may be linked to the death of newborn brain cells, which can disrupt learning, mood, and anxiety. The researchers found that a compound currently being developed could potentially prevent this cell death and have therapeutic value for these diseases.
Autologous cardiac cells, particularly atrial appendage-derived cells, show promise for gene and RNA therapies in heart failure. The review discusses financial and authority-based restrictions influencing research in this field, highlighting the potential of autologous tissues as cell therapy sources.
A biologically younger woman demonstrates the world's first successful gene therapy against human aging. Elizabeth Parrish's white blood cells showed a 20-year increase in telomere length, implying a reversal of age-related diseases.
A novel gene therapy developed at St. Jude Children's Research Hospital has shown promising results in rebuilding the immune system of young adults with X-linked severe combined immunodeficiency disease (SCID-X1). The treatment, which combines lentivirus gene therapy with busulfan conditioning, resulted in improved health and reduced c...
Researchers have discovered a novel host defense mechanism that protects cells from viral infection by sequestering virions near the nucleus. This discovery has implications for new drug discovery and therapeutic gene delivery, and may lead to improved strategies for rAAV-based gene therapy.
Dr. Crystal Mackall receives $500,000 Clinical Translation Grant to study osteosarcoma and develop immunotherapy treatment options. The grant aims to improve outcomes for patients with this rare form of cancer.
A new study in patients with methylmalonic acidemia found lower than expected levels of antibodies against adeno-associated viral vectors, suggesting encouraging implications for gene delivery as a treatment. The absence of AAV antibodies in most patients who lack the deficient enzyme has promising results for gene therapy.
Pediatric researchers identified a new syndrome causing intellectual disability, linked to a mutation in the TBCK gene. A potential targeted treatment using an amino acid supplement has been proposed based on cell studies.
Scientists at EMBL have increased the efficiency of gene therapy tool Sleeping Beauty, which is being trialed for therapies targeting B-cell leukaemias and lymphomas. The enhancement results in a 30% increase in efficiency and aims to improve patient outcomes.
A study published in JAMA Cardiology found that intracoronary gene transfer increased left ventricular function beyond standard heart failure therapy. The treatment, which involves introducing a specific protein into heart cells, showed significant benefits in patients with nonischemic heart failure.
Researchers have found that suppressing the nuclear receptor protein ROR-γ with small-molecule compounds can reduce androgen receptor levels in castration-resistant prostate cancer, stopping tumor growth. This novel approach targets the root cause of the problem - the overexpression of the AR gene and its protein.
A global study has identified hundreds of genes associated with 5 common inflammatory diseases, paving the way for new treatments. The research found that these conditions share similar genetic backgrounds and can occur together due to shared risk factors.
A new Scripps Research Institute study has identified specific genetic variations associated with increased susceptibility to bipolar disorder and other conditions. The research focuses on a gene called PDE10A, which produces proteins that regulate intracellular levels of cAMP, influencing biological processes like learning and memory.
Researchers discovered that healthy carriers of a rare respiratory disease gene have enhanced respiratory capacity and increased height, with a 10% advantage. The study may lead to new treatment approaches for physical fitness training and lung function disorders, as well as height modification in growth disorders.
Researchers present innovative approaches using cell transplantation and genetic engineering to address neurodegenerative diseases, including Alzheimer's and Huntington's. Studies show promise in reducing learning deficits and alleviating peripheral neuropathic pain, offering new hope for treatment options.
Researchers have identified two genes that, when altered, promote or undermine cardiovascular health. ANGPTL4 is associated with lower triglycerides and reduced risk of coronary disease, suggesting strategies to lower triglycerides could help prevent heart attacks. Future therapeutics targeting this pathway are in development.
Current gene sequencing methods struggle to decipher medically important genes in troublesome genome regions, leading to false positives and false negatives. Researchers call for more accurate sequencing to optimize medical care and prevent unnecessary surgeries.
Gene therapy has made significant progress in treating X-linked severe combined immunodeficiency (SCID-X1), with hematopoietic stem cells showing promise for a cure. However, ongoing challenges include improving safety and achieving long-term immune reconstitution, highlighting the need for continued research and development.
Researchers develop integrase-defective lentiviral vector to deliver chemotherapy-sensitizing gene to pancreatic tumor cells, reducing risk of insertional mutagenesis. The system enables high efficacy delivery of the gene that encodes for DCK protein to cancer cells, making them more sensitive to chemotherapeutic drug gemcitabine.
A team of researchers discovered that people with Down syndrome have altered white matter insulation in their brains, affecting nerve transmission. This finding may lead to new treatments for DS patients and has implications for other developmental disabilities like autism.
ESTEVE has developed two new investigational gene therapies, EGT-201 and EGT-301, to treat Sanfilippo B syndrome and Hunter syndrome. The treatments join a promising gene therapy platform aimed at restoring enzyme function in patients with severe and debilitating rare diseases.
Researchers have identified multiple gene therapy approaches to treat Alzheimer's disease, including boosting neuroprotection, increasing autophagy-related proteins, and regulating lipid metabolism. These strategies aim to address the underlying neuropathological changes associated with the disease.
Florida State University researchers are working on a new approach to deciphering genetic data that may lead to more targeted prostate cancer treatments. They identified altered signal pathways in prostate cancer, which could help pinpoint genetic changes that matter in cancer development and progression.
Scientists at the University of Sheffield have identified a network of genes that enables sharks to regenerate their teeth throughout their lifetime. Humans possess similar cells, but with limited tooth regeneration ability.
Researchers from Kumamoto University have identified the protein p53, which plays a crucial role in slowing down the progression of Alport syndrome. The study suggests that recovering the function of the p53 gene could help inhibit symptom progression and develop new treatment strategies for genetic diseases.
Researchers at Michigan Medicine have identified a strong interaction between KRAS and AGO2 that could contribute to its role in cancer development. The study suggests that targeting this interaction may be a potential therapy for KRAS-related cancers.
Researchers demonstrate selective gene delivery to modified upper motor neurons, showing promise for future gene replacement therapies. The study provides evidence that targets diseased cells with high specificity, laying the groundwork for effective treatment strategies.
A study found that HER2-positive lung cancer is caused by either gene amplification or mutation, rather than both. This distinction implies the need for different treatment options, including targeted therapies approved for breast cancer.
Researchers have pinpointed cell types responsible for common brain diseases, including Alzheimer's and Multiple Sclerosis. The findings suggest that developing medicines targeting microglial cells could offer hope for treating these conditions.
Researchers used CRISPR to repair a genetic mutation responsible for retinitis pigmentosa, an inherited condition causing blindness in at least 1.5 million cases worldwide. The study marks the first time researchers have replaced a defective gene associated with a sensory disease in stem cells derived from a patient's tissue.
Scientists have identified a protein called Tet3FL that acts as a 'guardian' against neurodegenerative diseases like Parkinson's. Tet3FL removes molecules called methyl groups from specific genes, keeping them active and maintaining cellular 'trash removal' processes.
Researchers discovered an abnormal gene expression in the lining of the womb that is associated with IVF failure. This finding could lead to a new test to predict treatment success and guide patient counseling, helping women understand their chances of achieving a pregnancy before starting treatment.
A University of Colorado study found distinct genetic drivers in colorectal cancer among young and old patients, with potential implications for targeted therapies.
Researchers have successfully used gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. The treatment showed long-term safety and efficacy, with treated dogs expressing therapeutic levels of clotting factor VII.
Researchers have identified three genetic associations that influence susceptibility to primary open angle glaucoma, a leading cause of irreversible blindness. The study found associations between TXNRD2, ATXN2, and FOXC1 genes, which may lead to the development of gene-based screening tests and therapy for glaucoma patients.
UCLA scientists have discovered a crucial role for the Rbfox1 gene in regulating genes that contribute to autism risk. The study highlights an untapped region in brain cells as a goldmine of drug targets for new treatments.
Researchers in gene therapy challenge a recent study linking adeno-associated virus 2 (AAV2) to liver cancer development, citing flawed experimental methods and data interpretation. AAV2 is found in up to 90% of humans, yet liver cancer affects only about 10/100,000 people in the US.
The Concise Guide to PHARMACOLOGY 2015/2016 provides an overview of 1,700 human drug targets, focusing on those exploited in the clinic or with future therapeutic potential. The guide combines evidence-based data with summaries of molecular targets, allowing users to rapidly gain insight into their function and comparative pharmacology.
Duke University researchers successfully treated an adult mouse model of Duchenne muscular dystrophy using CRISPR gene editing. The treatment involved delivering the gene-editing system directly to the affected tissues through a non-pathogenic carrier called adeno-associated virus, overcoming several delivery challenges.
Researchers at UTA have developed a new platform that uses ultrafast near-infrared lasers to deliver gene therapy to damaged areas of the retina, enabling vision restoration in patients with macular degeneration. The laser-based method has been shown to be more effective than traditional chemical gene delivery systems.
Researchers have identified 52 genetic variants associated with AMD, including associations between CFH and TIMP3 genes and the extracellular matrix. The study provides a framework for future studies of AMD biology and therapy development.
Researchers identified 52 genetic variations associated with AMD across 34 gene regions, offering hope for developing diagnostic tests and therapeutics. The study also found rare genetic variations in one subtype of AMD, which could explain why anti-VEGF therapy is less effective in some patients.
Researchers discovered that lung cancer in younger patients has distinct genetic subtypes that can be treated with available targeted therapies. However, these patients' survival times are shorter than expected due to the more aggressive nature of the disease.
A consensus panel of neuropathologists confirms CTE has a distinct pathognomonic signature in the brain, differentiating it from other tauopathies. The criteria define progressive degeneration of brain tissue, including abnormal protein buildup, leading to memory loss and dementia.
A new study has identified potential genetic alterations in penile cancer, revealing similarities with other squamous cell cancers. The researchers found a common combination of alterations in genes KRAS, HRAS, and NRAS, as well as EGFR, which may impact the tumor's response to an EGFR inhibitor.
Researchers at Houston Methodist Hospital developed a suicide gene therapy that combines radiation treatment with a genetically modified virus to target and destroy cancer cells. The treatment shows high five-year overall survival rates of 97% and 94%, improving upon historical studies by 5-20%.
A study found that breast cancer survivors with therapy-related leukemia often have inherited cancer susceptibility and gene mutations. Researchers suggest a long-term follow-up study to understand the impact of these genes on leukemia risk, enabling personalized conversations about treatment benefits and risks.
A new gene therapy platform uses a hydrogel and self-assembled nanoparticles to deliver microRNAs that suppress tumor tissue, increasing dosage at the site while reducing systemic side effects. The approach has shown promising results in shrinking tumors by nearly 90% in pre-clinical models.
Researchers report improvements in four children treated with gene therapy for Wiskott-Aldrich syndrome, including reduced bleeding events and improved immunologic symptoms. The treatment's long-term safety and efficacy remain to be assessed as the children are monitored for 15 years.