Articles tagged with Gene Therapy
The article considers the ethical issues surrounding enrolling children with neurodevelopmental conditions, such as autism spectrum disorder and fragile X syndrome, in clinical trials. Parents may face difficult decisions about whether to enroll their children due to concerns about potential loss of positive aspects of their condition.
A study by Duke University researchers found antibiotic-resistant bacteria in the guts of lemurs living close to humans. The closer the contact, the more resistant bugs were found. Proximity to humans determined the type and abundance of resistance genes acquired.
Researchers from the Hubrecht Institute have successfully corrected mutations that cause cystic fibrosis in cultured human stem cells using prime editing. The technique is safer than CRISPR/Cas9 and shows promise for application in patients, potentially leading to a cure or prevention of genetic diseases.
Researchers discovered a new way to treat PTSD by targeting epigenetic modifications. Combining two natural products, SAMe and vitamin A, reversed PTSD-like behaviors in animals. This approach offers hope for a non-toxic treatment that addresses the underlying genetic cause of the disease.
A study published in Trends in Genetics reveals that the cat genome is remarkably similar to the human genome, with similarities found even in mice and dogs. This discovery holds promise for understanding genetic diseases in cats and humans, including polycystic kidney disease and hypertrophic cardiomyopathy.
Researchers at Children's Hospital of Philadelphia have developed a system that can fine-tune protein expression from gene therapy vectors, addressing the need for controlled dosing. The 'dimmer switch' uses alternative RNA splicing and an orally available small molecule to adjust levels of expression up or down as needed.
A large-scale genetic study has identified new drug targets to treat type 1 diabetes by examining 61,427 participants and pinpointing 78 regions on the chromosomes that influence risk. The researchers found 36 previously unknown regions and specific gene variations that can be used to identify potential drug targets.
Scientists discover gene therapy that protects optic nerve cells and preserves vision in mouse models of glaucoma. The study suggests a way forward for developing neuroprotective therapies for glaucoma, a leading cause of visual impairment and blindness.
Scientists discovered that cells from different organs are differentially susceptible to activating mutations in cancer drivers, leading to distinct outcomes. The findings highlight the importance of understanding tissue-specific genetic networks and interactions to develop precise molecular interventions.
Scientists found that HtrA1 protein levels decrease with age in individuals with chromosome 10 risk variants associated with age-related macular degeneration. The study identified HtrA1 as a protective factor, suggesting it maintains the integrity of the RPE-Bruch's membrane interface.
A new study suggests that gene therapy delivered into the brain may be safe and effective in treating AADC deficiency. The therapy resulted in clinical improvement of symptoms, including reduced oculogyric crises, improved movement and motor function, and better sleep patterns.
A novel gene therapy method has shown dramatic improvement in symptoms, motor function, and quality of life for children with AADC deficiency. The treatment involves introducing a benign virus programmed with specific DNA into precisely targeted areas of the brain.
A comprehensive genome-wide association study identified 13 loci strongly associated with infection or severe COVID-19, including causal factors like smoking and high body mass index. The findings could provide targets for repurposed drugs and illustrate the power of genetic studies in infectious disease.
A new study reveals that light therapy's benefits come from activating the Period1 gene in a brain region involved in mood regulation. Exposure to light at the end of the dark period has an antidepressant effect on mice.
A global initiative involving Qatar Foundation Research has identified 13 loci associated with severe COVID-19 severity. The study highlights the importance of diversity in genetic datasets and paves the way for future therapeutic targets.
A team at Tokyo Medical and Dental University identified four proteins that bind to Toc-HDOs, regulating gene silencing. The discovery provides a novel biological mechanism for Toc-HDOs, increasing understanding of how they silence genes.
Researchers at Mayo Clinic have discovered a single-gene cause of a neurodevelopmental disorder, identifying 28 unique variants in the SPTBN1 gene. The study provides hope for diagnosis and potential treatment for affected individuals.
Researchers at Baylor College of Medicine have developed a novel gene therapy that shows promise in treating human heart failure. The treatment targets the Hippo signaling pathway, which can inhibit heart repair, and has been shown to improve heart function and promote tissue renewal in pig models after a heart attack.
Scientists at UCL GOS ICH and Great Ormond Street Hospital have developed a precision gene therapy that has been shown to relieve defects in DTDS brain cells, potentially offering a cure for the condition. The therapy was tested on laboratory models and mice, showing promising results and paving the way for a clinical trial.
Researchers have linked specific genes to obsessive-compulsive disorder (OCD) using genome-wide analysis, pointing toward novel avenues for treatment. The study identified a strong correlation between OCD and rare mutations in genes such as SLITRK5.
A new multiplex technique developed at Goethe University Frankfurt allows simulating millions of genetic defect combinations and studying their effects in cell culture. This enables the identification of genes involved in cancer and other complex diseases suitable as targets for therapies.
An international study on rare childhood cancer, rhabdomyosarcoma, found that mutations in genes TP53, MYOD1, and CDKN2A are associated with a more aggressive form of the disease. The study provides genetic clues for developing targeted therapies.
Researchers have identified neural crest stem cells as a key factor in non-genetic drug resistance mechanisms, which can help predict potential resistance routes in patients and develop personalized therapies.
Researchers at UNC School of Medicine developed an improved oligonucleotide therapy strategy that can correct gene defects underlying cystic fibrosis. The approach has shown promising results in both human cells and mice, with potential applications for treating other pulmonary diseases.
A convolutional neural network-based algorithm predicts tumor mutational burden-high (TMB-H) colorectal cancer response to immunotherapy. The model uses histopathological characteristics, particularly tumor-infiltrating lymphocytes, from hematoxylin and eosin-stained slides.
Researchers developed a novel gene therapy approach that utilizes acetaminophen to correct disease-causing mutations and make liver cells immune to its toxic effects. This allows treated cells to multiply and reduces the need for liver transplants in treating genetic disorders.
Researchers have developed a platform that produces bespoke star-shaped polypeptide nanoparticles, effectively delivering range of therapies including gene therapies. The material accelerates bone tissue regeneration with a six-fold increase in new bone formation.
A preclinical study supports AAV8-TNAP-D10 as a single-dose treatment for hypophosphatasia, extending the life span of mice with minimal side effects. The therapy may provide an alternative to current medication injections, benefiting patients with severe skeletal and dental abnormalities.
The Treatabolome project is an EU initiative that seeks to deliver rare disease and gene-specific treatment information to healthcare professionals. Systematic reviews on various rare neurological disorders are now available, providing a foundation for the platform's database.
The acylfulvene alkylating agent LP-184 demonstrates highly potent anticancer activity in NSCLC cell lines, correlating with PTGR1 transcript levels. It targets tumors regardless of co-occurring mutations but is especially effective in KEAP1 mutant settings.
Researchers have found that a truncated version of the otoferlin gene, including its transmembrane domain, is essential for proper neurotransmitter release and sensory cell maturation. This discovery brings scientists closer to developing gene therapy treatments for severe congenital hearing loss.
A Salk study reveals the connection between CRTC3 and melanin production, finding that eliminating the protein can reduce melanoma cell aggression. The researchers also discovered two cellular communications systems converge on CRTC3, suggesting it as a potential target for developing new treatments.
University of Maryland faculty members Shelby Bensi, Gregg Duncan, Katrina Groth, and Katharina Maisel have received CAREER grants for their innovative research in hazard assessment, gene therapy, risk assessment, and immunotherapy. Their work aims to advance the fields of civil engineering, bioengineering, and mechanical engineering.
Researchers have created nanoparticles that can effectively deliver and protect siRNA, a promising class of therapeutics targeting harmful genes. This breakthrough aims to improve the treatment of diseases by fine-tuning mRNA production, offering a new approach to managing irregular protein expression.
Researchers developed a gene therapy that successfully treated 48 out of 50 children with ADA-SCID, a rare life-threatening disease. The treatment involves removing stem cells, delivering a new copy of the ADA gene, and returning the corrected cells to produce healthy immune cells.
A new form of gene therapy has successfully treated 48 out of 50 children born with a rare and deadly inherited disorder. The treatment, developed by an international team, involves delivering a corrected copy of the ADA gene into stem cells, which are then returned to the child's body to produce healthy immune cells.
Infants and children with ADA-SCID, a life-threatening inherited immunodeficiency disorder, have regained immune system function after receiving gene therapy. The treatment involves inserting a normal copy of the ADA gene into blood-forming stem cells, offering a one-time procedure with long-term benefits.
Scientists at the University of Colorado Anschutz Medical Campus discovered that intense light can enhance the expression of the ANGPTL4 gene, which regulates fatty lipids in plasma. This could lead to a new tool for safely blocking the gene and reducing cardiovascular risk.
Nanoscope Technologies is featuring groundbreaking research on optical gene delivery and functional characterization platform technologies for treating retinal diseases. The company's lead product, vMCO-010, delivers light-sensitive Multi-Characteristics Opsin into retinal cells to restore vision in patients with degeneration.
Researchers used gene therapy to introduce a therapeutic compound into the hippocampus of AD mice, preserving learning and memory. The approach restored normal neural function and reversed neurodegenerative processes by promoting neuronal and synaptic plasticity.
CRISPR/Cas technology has advanced diagnostics and gene therapy, enabling fast and accurate disease diagnosis and treating untreatable diseases like cancer and blood disorders. Recent advances in CRISPR-Cas technologies are highlighted, with a discussion of the strength and challenges for its future clinical usage.
Researchers developed a new approach called Co-opting Regulation Bypass Repair (CRBR) that can correct genetic diseases caused by single gene mutations. The method uses the CRISPR/Cas9 system and non-homologous end joining to insert a functional copy of the gene, enabling treatment in all adult tissues.
A Monash University study has discovered a way to prevent and reverse the damage caused by broken-heart syndrome, also known as Takotsubo cardiomyopathy. The breakthrough uses Suberanilohydroxamic acid to target genes and improve cardiac health.
Researchers at Penn Medicine delivered a new gene therapy that improved vision in three patients with severe vision impairments. The therapy showed sustained improvements in day and night vision, without serious side effects.
Researchers at Wake Forest School of Medicine have identified a set of new genetic markers that could lead to targeted treatments for non-small cell lung cancer. The study found that tumors with mutated KMT2 genes exhibit genetic instability, making them more sensitive to PARP inhibitors.
The Alliance for Cancer Gene Therapy Summit 2021 features key findings on translating CAR T-cell therapy success to complex solid tumors. Researchers share insights on evolving cell and gene regulatory processes, innovative science from the lab to the clinic, and tackling challenges in pancreatic cancer and glioblastoma.
A gene therapy protects eye cells in mice with a rare disorder, suggesting a combination approach may preserve vision in people with retinitis pigmentosa. Researchers found that using Txnip gene therapies, along with treatments for oxidative stress and inflammation, provided additional protection for the cells.
A new study expands evaluation of gene therapy for spinal muscular atrophy (SMA), a rare genetic neurodegenerative disease. Treatment with onasemnogene abeparvovec is associated with an immune response, but showed no long-term adverse effects.
Researchers discovered a cell-to-cell communication network that instructs tumor cells in tissues to regrow after genotoxic therapy. The study found that elevated p53 levels and release of interleukin-6 signal cells to activate growth signals, leading to tumor regrowth.
The study shows that using a truncated form of the CD4 molecule as part of a gene therapy yields superior and longer-lasting results in mouse models than previous similar therapies. The new approach to CAR T gene therapy has the potential to create lifelong protection from HIV infection by producing memory cells.
Researchers at Penn Medicine have developed an RNA therapy that reversed blindness in a patient with a rare genetic disorder, improving vision over a 15-month period after a single injection. The treatment's durability and effectiveness provide new avenues for treating other ciliopathies.
Scientists at the University of Cambridge have demonstrated a new approach to gene therapy that may help repair damage caused by neurodegenerative conditions like glaucoma and dementia. The study, published in Science Advances, shows promise for treating chronic degenerative diseases.
A new siRNA delivery system allows for in vivo self-assembled small RNA synthesis, enabling potent target gene silencing in various diseases. This strategy overcomes the limitations of conventional delivery methods, offering a promising approach to RNAi therapy.
A new gene therapy approach has been successful in restoring both normal structure and function to the retina's cone photoreceptor cells in dogs with a severe form of Leber congenital amaurosis. The treatment, which delivered a normal copy of the NPHP5 gene, was tested in nine five-week-old dogs and showed promising results.
Researchers have discovered 12 genetic regions linked to lacunar strokes, which can lead to long-term disability and dementia. The study provides new hope for preventing and treating this devastating type of stroke.
A new study evaluates the economic impact of gene therapy on severe sickle cell disease, suggesting substantial costs and potential cost savings for Medicaid programs. The analysis estimates a high prevalence of sickle cell disease among these programs, which may affect treatment affordability.
A new gene therapy approach using zinc finger proteins has shown long-lasting effects in reducing tau protein levels and reversing brain damage in an animal model of Alzheimer's disease. The treatment involves a single injection and could potentially lead to a new treatment for patients with this devastating illness.
A study found that gene therapy can create a long-term store of correct T cells in the human thymus, leading to sustained health in patients with SCID-X1. The treatment works by delivering corrected genes into stem cells, which then produce healthy immune cells.
Researchers have identified a new blindness gene, IFT122, associated with inherited retinal dystrophy in dogs. The discovery has significant implications for understanding the genetic background of the disease and developing novel treatments.
A team led by Dr. Brian Brown is developing a CAR T-cell therapy that specifically targets immune-suppressing macrophages in tumors, clearing the way for other T cells to destroy cancer cells. The grant aims to advance this promising approach and bring new hope to patients with solid tumor cancers.