Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Scientists at the University of Granada have developed a new therapy for skin and lung cancer using a suicide coliphage-gene, demonstrating effectiveness in vitro and in vivo. The treatment involves inducing cell death in tumour cells, potentially reducing the need for chemotherapy.
A Phase 1/2 study of LentiGlobin gene therapy has demonstrated positive results in a young adult with severe beta-thalassemia, achieving transfusion independence for over two years. The treatment also identified cells with the corrected beta-globin gene that overexpressed HMGA2.
Researchers found that inhibiting GRK2 protects heart cells from destruction after a heart attack, while excess GRK2 leads to more damage. The discovery suggests that anti-GRK2 gene therapy may help prevent permanent heart damage.
Researchers are calling for a new focus on hypertrophic cardiomyopathy (HCM), a genetic disorder causing sudden death in young people. A task force of cardiologists and cardiac biologists recommends studying the natural history of HCM, defining all genetic causes, supporting clinical trials, and preventing mutant gene expression.
Scientists at Duke University Medical Center have identified a gene associated with myopia in Caucasian people, providing new hope for gene therapies. The RASGRF1 gene plays a crucial role in retinal function and visual memory consolidation, making it a promising target for treatment.
A new study reveals that CEP290 is crucial for maintaining the structural integrity of the ciliary gate, a key component of cilia. This discovery could lead to targeted gene therapy for cilia-related disorders, including Meckel syndrome and Joubert syndrome.
A multidisciplinary team at UCLA has successfully visualized a virus using cryo-electron microscopy, enabling the adaptation of the adenovirus for use in gene-therapy treatments for prostate and breast cancers. The breakthrough provides critical structural information for researchers attempting to modify the adenovirus.
A team of 64 international researchers has identified 27 new MiniPromoters, greatly expanding the current limited pool of these vital tools. The Pleiades Promoter Project enables research worldwide with bioinformatics, genomics, and neuroimaging expertise.
Researchers at Scripps Research Institute have determined the atomic structure of a human adenovirus, leading to insights into its assembly and potential applications in gene therapy. The largest complex ever solved at atomic resolution, this discovery may lead to more effective treatments for diseases such as cystic fibrosis and cancer.
Researchers found a genetic rarity where disease-causing genes can self-repair, resulting in spots of normal skin amidst diseased skin. The recombination of chromosomes prior to cell division leads to cells with either two mutant copies or no mutant copies, forming normal skin patches.
A recent study by UCLA researchers has identified a genetic variation linked to systemic lupus erythematosus (SLE) in Asian men. The findings suggest that this variation on the X chromosome enhances an immune response, increasing the risk of developing lupus in men compared to women.
Researchers at Tufts University develop nanoparticles to deliver therapeutic genes to the retina, delaying onset of eye disease and preserving vision. The treatment, using a gene for GDNF, shows temporary but significant protection against photoreceptor cell death.
Researchers successfully replaced a human gene to preserve photoreceptor function in mice with severe retinal degeneration. The study demonstrates improved function and survival of photoreceptors, validating a potential gene therapy design for patients affected by Leber congenital amaurosis.
Scientists have developed a new approach to deliver therapeutic genes into brain cancer cells, using chlorotoxin from deathstalker scorpion venom and nanoparticles of iron oxide. This method has shown promise in inducing nearly twice the amount of gene expression in brain cancer cells compared to traditional approaches.
Gene therapy holds promise in treating prostate cancer, but challenges remain in achieving effective treatment. Researchers call for better testing systems and innovative approaches to advance the field. Prostate cancer is a leading cause of cancer death among men in the US.
Italian scientists have successfully completed preclinical trials for a gene transfer treatment that can correct the lack of beta-globin in patients' blood cells, a major step towards treating beta-thalassemia. The treatment uses genetically corrected stem cells to restore haemoglobin production and overcome the disorder.
Researchers at Biogen Idec Inc. have found that targeting the TIM-1 protein may be effective in treating asthma. The study used a humanized mouse model to show that an antibody binding to a specific region of TIM-1 reduced inflammation and airway hyperresponsiveness.
A genetically reprogrammed herpes virus and an anti-vascular drug combination shrinks spreading distant sarcomas in mice, with significant tumor reduction and improved survival rates. Researchers achieved these results by using the virus systemically via injection, overcoming a major stumbling block in previous cancer treatments.
Researchers create nanoparticle that effectively delivers genetic material into cells, outperforming existing methods. The device, a vector, is between 2 ½ and 10 times more effective than other experimental materials, according to the research.
A personalized approach to smoking cessation is developing, combining genetic information and smoking habits to predict effective nicotine replacement therapy. This method could increase cessation success rates by up to 25%.
Researchers developed an experimental cure for Type 1 diabetes using gene therapy, which successfully reversed the disease in about half of nonobese diabetic mice. The treatment protected new beta cells from autoimmune attack by adding a protective gene, allowing them to function normally.
A study found that patients with PTSD who carried a short allele of the serotonin transporter gene promoter responded more poorly to cognitive behavior therapy. The researchers suggest that genetic variation may influence treatment response, leading to potential modifications in treatment approaches.
Researchers at the University of Montreal have identified the mechanism behind three cancer-causing genes in acute lymphoblastic leukemia. The study provides insight into the complex interaction between these genes and their contributions to leukemia, which could lead to the development of less invasive cancer therapies.
Research on prion disease found that structural differences affect transmission between species, while gene therapy showed promise in nonhuman primates. Additionally, the protein periostin was discovered to promote heart valve degeneration by inducing angiogenesis and MMP production.
A small genetic change in the IL28B gene can predict how well individuals with hepatitis C will respond to treatment. Those carrying this protective allele are more likely to clear the virus and respond to therapy.
Researchers at the University of Oklahoma Health Sciences Center have found a way to use gene therapy to treat retinitis pigmentosa, an eye disease that causes blindness. The treatment involves delivering genetic therapies directly to light-sensitive cells in the retina using nanoparticle technology.
Researchers at the University of Sheffield have developed a novel gene transfer system that can restore SMN protein levels and alleviate symptoms in SMA model mice. The new technique has the potential to develop a simple injection therapy without surgical interventions, offering significant implications for future SMA treatment.
A new gene therapy called SERCA2a has been shown to be safe and effective in reversing advanced heart failure by stimulating the production of an enzyme that enables the heart to pump more effectively. Patients who received SERCA2a demonstrated improvements in symptoms, heart function, and severity of heart failure.
Researchers at McGill University have discovered a way to control the normal expression of genes, including those active in cancer, through RNA interference. This breakthrough has important applications for laboratory research and could lead to the development of individually tailored cancer therapies.
Researchers found that increasing BMPR2 levels reduced pulmonary hypertension and strain on the heart in rat models of the disease. The study suggests that boosting BMPR2 levels could offer a new therapeutic target for treating PAH.
A consistent pattern in five genes has been found to predict the likelihood of relapse in pediatric T-cell acute lymphoblastic leukemia (T-ALL), allowing for more aggressive treatment to be administered from diagnosis. This discovery has significant potential to improve outcomes for patients at high risk of relapse.
A genetic link has been discovered between sporadic and familial forms of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease. The study found that protein FUS forms characteristic inclusions in spinal motor neurons in most ALS cases, suggesting a common pathogenic pathway for motor neuron degeneration.
Veterinary scientists at Penn have successfully used a viral vector to target rod cells in the retina, a critical step towards developing gene therapies for inherited blindness. The study demonstrates the potential of this approach to correct diseases such as X-linked and autosomal dominant retinitis pigmentosa.
Researchers at Penn University successfully used gene therapy to restore retinal cone function and day vision in canines with inherited day blindness. The treatment was effective for younger dogs and held promise for future clinical trials.
A team of researchers from Université Laval has proven that it is possible to repair the defective gene responsible for Duchenne muscular dystrophy. They developed enzymes called meganucleases that can correct the dystrophin gene and restore its expression in muscle cells.
An international expert group recommends individualized treatment for patients with type 2 diabetes, citing the need to address underlying physiological and genetic differences. The recommendations aim to improve glycemic control, minimize risk, and reduce diabetes complications by tailoring therapies to specific patient subgroups.
Researchers found that only patients with a specific genetic mutation are eligible for gene therapy, which has shown promising results in treating Leber congenital amaurosis. Genetic testing can detect changes in the genome, but accurate interpretation is crucial to ensure correct treatment.
Scientists have successfully used gene therapy to restore vision in mice with retinitis pigmentosa, a degenerative eye disease. The treatment involved the use of compacted DNA nanoparticles, which improved structural and functional vision in affected mice, without any adverse effects.
The median survival time for metastatic breast cancer patients increased steadily over 25 years, with improved outcomes for those with grade III tumours. Targeted therapies, particularly trastuzumab, have contributed to these advancements, offering new hope for patients with aggressive forms of the disease.
Researchers at Cedars-Sinai Medical Center have identified over 30 regions of the genome linked to ulcerative colitis, providing insights into the disease's causes and potential new therapies. This understanding may lead to more effective treatments tailored to individual patients' needs.
A new gene therapy approach has shown promising results in treating spinal muscular atrophy (SMA) in mice by improving muscle strength, coordination, and locomotion. The treatment involves injecting the gene-carrying therapeutic directly into the brain and spinal cord of newborn mice.
Christopher Evans, a leading molecular biologist, has received the prestigious Arthur Steindler Award for his significant contributions to understanding musculoskeletal diseases. His innovative gene therapy work holds great promise as a new treatment option for managing rheumatoid arthritis and osteoarthritis.
Researchers at Universite de Montreal and VGTI have discovered a new mechanism by which HIV infects immune cells, characterizing the role of two molecules PD-1 and IL-10 in this process. The study suggests that blocking these interactions may restore the immune response in HIV-infected patients.
A second dose of gene therapy has proven safe and effective in animal studies, suggesting that patients who benefit from treatment in one eye may experience similar benefits in the other. The study found no signs of toxic side effects or interference from unwanted immune reactions.
Researchers used gene therapy to deliver a portion of DNA that makes the SMN protein into newborn mice, resulting in improved muscle coordination and longer survival. The treatment corrected motor function, restored nerve signals, and improved overall health in mice with spinal muscular atrophy.
Fetal surgery continues to evolve with advancements in prenatal diagnosis and surgical techniques. Experts at the Children's Hospital of Philadelphia describe innovative procedures for treating rare defects, including lung masses, sacrococcygeal teratomas, and spina bifida.
Physicist Alex Evilevitch directly measured the energy associated with viral DNA expulsion, a discovery that could lead to broad-spectrum antiviral drugs. The study used isothermal titration calorimetry and found that increasing DNA length increases heat release, highlighting the importance of hydration entropy in viral genome packaging.
A new study by the University of Pennsylvania School of Medicine found that extended use of a nicotine patch improves abstinence from cigarettes and helps smokers quit again after relapse. The extended therapy regimen was more effective in boosting abstinence rates than standard therapy.
A new tool called PEG-POD significantly increases gene delivery to retinal cells, outperforming existing carriers and DNA alone. This breakthrough may help develop therapies for conditions like retinitis pigmentosa and age-related macular degeneration.
University of Florida researchers are developing a gene therapy to treat Pompe disease, which causes severe muscle weakness and breathing difficulties. The treatment involves delivering a corrective gene directly to the diaphragm using an adeno-associated virus, with clinical trials expected to begin soon.
Researchers have achieved a breakthrough in regulating genes by hijacking riboswitches, opening doors for targeted drug discovery and synthetic biology. The study, published in Proceedings of the National Academy of Sciences, uses synthetic molecules to activate genes previously controlled by small naturally occurring molecules.
Researchers have identified a human genetic alteration associated with anxiety-related behavior in both humans and mice, which can inform the development of new clinical strategies to treat anxiety disorders. The study used functional magnetic resonance imaging (fMRI) to reveal altered brain function in individuals with this abnormality.
Researchers found that gene therapy to express a constitutively active form of protein I-1c in heart muscle cells improved contractile function in young mice, but led to abnormal heartbeats and sudden death under stress. Older mice developed characteristic features of heart failure after treatment.
Neuroscientists combined herpes and Sleeping Beauty to deliver larger genes to brain cells, increasing the size of genetic payload by nearly tripling it. This breakthrough technology could lead to treatments for devastating brain diseases like pediatric leukodystrophies and lysosomal storage disorders.
A study published in Cancer Cell reveals that epigenetic differences can distinguish patients with acute myeloid leukemia (AML) into subtypes with varying responsiveness to therapy. A set of 15-gene DNA methylation biomarkers was found to be highly predictive of patient survival.
Researchers from Boston University School of Medicine have discovered a gene therapy that can prevent the progression of emphysema by delivering therapeutic genes to lung tissue. The treatment achieved sustained expression of human alpha-1 antitrypsin protein, improving lung compliance and alveolar size in mice with emphysema.
An international team of researchers has identified a gene associated with DFN2, a rare form of progressive deafness that primarily affects males. The discovery offers therapeutic implications and may lead to treatments for other types of deafness.
Researchers at German Cancer Research Center find that HIV virus rarely integrates into genes with high transcription activity. This discovery could lead to the development of specific AIDS drugs targeting these 'cold spots', potentially offering a new approach to combat the virus.
Researchers identified specific genetic changes associated with an increased risk of relapse in cervical cancer patients. These changes are crucial steps in the progression of the disease towards an aggressive and treatment-resistant state.
Researchers have successfully transplanted genetically modified hematopoietic stem cells into mice, allowing their developing red blood cells to produce a critical lysosomal enzyme and preventing or reducing organ and central nervous system damage from Hurler's syndrome. This approach has the potential to improve treatment options for ...