Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
A recent study by Celmatix Inc. suggests that women may be stopping IVF treatment prematurely, with 68% conceiving within two cycles and over half (60%) becoming pregnant if they proceeded with an additional two cycles. The study also explored genetic markers for female infertility and IVF success.
Researchers at the University of Pennsylvania have made an encouraging breakthrough in developing a gene therapy to treat macular dystrophy. They successfully delivered a healthy copy of the BEST1 gene to dogs using viral vectors, showing promise for preventing or restoring vision in individuals with these conditions.
African-American individuals are disproportionately affected by glaucoma, with a four to five times higher risk of occurrence and up to 15 times greater likelihood of significant visual impairment compared to European descent.
Researchers have identified a genetic target, BCL11A enhancer, that controls hemoglobin production in red blood cells. This discovery opens the door to precise targeting of sickle cell disease with potential benefits without off-target effects.
A new gene transfer strategy using modified human stem cells introduces chimeric antigen receptors to target tumor cells, leading to a persistent anti-tumor immune response. This approach has shown promise in treating blood cancers such as leukemia and lymphoma.
Scientists have redefined the MECP2 protein's role in Rett syndrome, discovering it acts as a global activator rather than repressor. This new understanding can lead to novel therapies for the disease, targeting the AKT/mTOR pathway to reverse symptoms.
A team of scientists has developed a process to connect an autism-linked gene to its function. They found that mutations in the NHE9 gene cause communication problems among brain cells, contributing to autism. The study used a step-wise strategy to screen variants and identified changes that affect endosomal pH, altering cell traffic.
The study identifies syndecan-1 as the human cell receptor used by baculovirus to enter cells, shedding light on the mechanisms of infection and facilitating gene therapy development. This breakthrough discovery increases our understanding of baculovirus interactions with human cells.
A study led by Dr. Luca Scorrano and Dr. José Antonio Enríquez found that the OPA1 gene can regulate cellular metabolism, potentially leading to new treatments for mitochondrial diseases. The researchers discovered that increasing OPA1 activity enhances energy production and cell growth.
A new trial will test an anti-amyloid treatment in 650 adults at highest genetic risk for late-onset Alzheimer's, using the Alzheimer's Prevention Registry for enrollment. The study aims to prevent clinical onset of Alzheimer's and find effective treatments as soon as possible.
The National Cancer Institute has awarded a five-year, $11.3 million competitive grant renewal to Fred Hutchinson Cancer Research Center to lead the Pacific Northwest Prostate Cancer SPORE consortium. The consortium aims to unravel molecular mechanisms and develop precision-medicine approaches for prostate cancer treatment.
A new therapy has shown promising results in slowing the onset and progression of Lou Gehrig's disease, increasing survival rates by up to 39% in animal models. The treatment targets the SOD1 gene, which is mutated in some cases of familial ALS, and was administered via non-invasive delivery.
A novel machine learning-based approach predicts hepatotoxic potential of antisense oligonucleotides based on chemical sequence. The method achieved 74% accuracy in predicting toxicity and was used to redesign a therapeutic oligonucleotide with reduced potential for liver harm.
Researchers at the University of Leicester have discovered a potential molecular defence against Huntington's disease, a fatal neurodegenerative disorder. Glutathione peroxidase activity was found to be robustly protective in models of the disease.
Researchers have developed a new gene therapy approach to convert fibroblasts from human fetal heart cells and skin into heart muscle cells. This technique may potentially treat heart disease by regenerating a healthy heart within the damaged one.
A recent study found that children with amplified chromosome 21 are at higher risk of treatment failure, but not worse outcomes, than those without the genetic abnormality. Recognizing this amplification could lead to improved cure rates in low-risk patients treated aggressively.
A new gene therapy study, published in the Journal of Neuroscience, shows reversal of Rett symptoms in fully symptomatic mice by delivering a healthy MECP2 gene to cells throughout the body and brain. The treatment improved motor function, tremors, seizures, and hand clasping in 65% of cells.
A study published in the American Journal of Human Genetics found that whole-genome sequencing can identify genetic mutations associated with chronic mountain sickness. Researchers sequenced the genomes of Andean individuals and identified two genes, ANP32D and SENP1, which play a key role in hypoxia tolerance.
Researchers have found that specific DNA sequences in enhancers affect gene activity, enabling the design of synthetic DNA for gene therapy. This discovery may advance efforts to treat diseases using gene and cell therapies.
Researchers at Rice University have made strides toward creating a set of rules to design custom viral capsid proteins for gene therapy. By combining pieces of different adeno-associated viruses, they can create new, benign viruses that deliver DNA payloads to specific cells. This research has the potential to treat diseases such as ge...
A large-scale international study has identified 25 new mutations on nine key genes behind severe childhood epilepsy, including two new genes never before associated with the disorder. The findings suggest a new direction for developing genome-wide diagnostic screens and precise therapies for the condition.
Researchers have successfully combined two promising gene therapies to repair muscle damage caused by Duchenne muscular dystrophy and prevent future injury. The treatment, which uses a combination of micro-dystrophin and follistatin, showed significant increases in muscle strength and reduced muscle damage in aged mice.
A study by Professor Lesley Campbell and Dr Arthur Jenkins reveals that rare genetic defects are responsible for most of the problem with obesity and type 2 diabetes. The researchers found that many genes are involved, each contributing a unique genetic story, making it difficult to develop a single anti-obesity drug or treatment.
A study published in Cell reveals the key role of bromodomain and extraterminal domain proteins in activating genes contributing to heart failure. BET-inhibiting drugs show promise as a new treatment avenue.
Katherine A. High, MD, receives the 2013 E. Donnall Thomas Lecture and Prize for her pioneering research on gene therapy for hemophilia and inherited retinal degenerative diseases. Her work has led to sustained clinical improvements using adeno-associated virus vectors.
Researchers have shown that inhalable gene therapy can completely reverse pulmonary arterial hypertension in rat models and reduce expression of SERCA2a, an enzyme critical for calcium pumping. This approach may offer a promising therapeutic intervention for PAH patients.
Scientists discovered a molecular switch that regulates regeneration in flatworms, enabling them to grow heads complete with brain, eyes, and wiring. This breakthrough could lead to insights into why some animals regenerate while others don't, potentially informing regenerative therapies for humans.
Researchers from the University of Manchester have developed a new stem cell gene therapy to treat Sanfilippo, a fatal genetic brain disease. The treatment has shown promising results in mice, producing near-normal levels of SGSH enzyme and correcting progressive dementia and hyperactivity.
A team led by Fred Hutchinson Cancer Research Center scientists is developing precision therapies that selectively kill cancer cells while sparing normal tissue. Using high-throughput screening, they aim to identify new genes to target that may be highly specific to each patient's tumor.
Researchers have developed a bioengineered decoy that fools the immune system and prevents it from neutralizing the benefits delivered by a corrective gene. The approach could potentially increase the number of patients who can be treated with gene therapy, offering new hope for genetic diseases like hemophilia.
Researchers have identified key regions in the dengue virus genome that can be targeted to prevent replication. Artificial microRNAs were constructed to specifically target these sites, leading to effective inhibition of viral replication in humans. This innovative approach offers new hope for treating millions affected by dengue fever.
Gene therapy vectors derived from HIV successfully treat metachromatic leukodystrophy and Wiskott-Aldrich syndrome, restoring missing protein and reversing neurodegenerative process. The treatment is safe and effective, offering hope for patients with severe genetic diseases.
A new gene therapy approach using a partially inactivated lentivirus has shown significant improvement in three children with Wiskott-Aldrich Syndrome. The therapy corrects the genetic defect in blood cells by introducing a normal WASP gene, reducing the risk of cancer-promoting genes.
Researchers at Duke University Medical Center developed a blood-based test that accurately identifies who will respond to aspirin therapy and predicts patients at risk for heart attacks. The test, known as the 'aspirin response signature,' measures gene activity patterns in the blood.
A new gene therapy treatment has been developed to cure Sanfilippo Syndrome type A in animal models by introducing the sulfamidase enzyme into cells, eliminating glycosaminoglican accumulation and associated neuroinflammation. The treatment has shown promising results in mice and dogs, with improved behavior and life expectancy.
Researchers have made significant progress in developing new gene therapies to treat Sickle Cell Disease, malaria, and Mucopolysaccharidosis Type IIIA. In the Journal of Clinical Investigation, scientists successfully delivered a replacement gene to the brain in mice and dogs with MPSIIIA using intra-cerebrospinal fluid gene therapy.
At least half of birth-related deafness and many progressive hearing losses have a genetic basis. New sequencing technologies identify 1,000 mutations linked to hearing loss in 64 human genes. This knowledge will lead to practical treatments and effective genetic counseling.
A gene therapy virus has been approved for treating a rare inherited metabolic disease, and researchers found it rarely integrates into the genome, making it safe. The virus was analyzed in patients and mice, revealing its ability to target mitochondria and potentially correcting genetic defects in human mitochondrial DNA.
Researchers develop an engineered virus that can deliver genes to difficult-to-reach cells in the retina, restoring vision to patients with inherited defects and degenerative illnesses. The new therapy is surgically non-invasive and takes only 15 minutes to administer.
Researchers have identified the TTC7A gene as the cause of multiple intestinal atresia (MIA), a rare and life-threatening condition. The discovery will enable the development of prenatal diagnostic tests for pregnant women and screening tests for carriers, offering new hope for affected families.
A new study found that patients with PTSD who have a less functional variant of the BDNF gene respond poorly to exposure therapy. The study suggests that BDNF levels modulate the therapeutic effects of treatments for mood and anxiety disorders.
University of Michigan researchers have discovered how a defective gene in Down syndrome is regulated and its impact on neurological development. By studying fruit fly neurons, they identified two molecular pathways that converge to regulate the gene's abundance, offering a possible therapeutic approach to an aspect of the syndrome.
Researchers at Duke University have developed a novel genetic editing technique that repairs faulty genes responsible for Duchenne muscular dystrophy. The approach, which uses artificial enzymes to modify gene sequences, has shown promise in treating the disease, with over 60% of patients potentially benefiting from this new method.
Scientists use antigen-decorated nanoparticles to prevent immune over-reaction in mice, while also developing a potential gene therapy for Mucopolysaccharidosis Type IIIA. Meanwhile, researchers discover a new target for castration-resistant prostate cancer by blocking mutant androgen receptors.
A subset of metastatic colorectal cancers responds to anti-EGFR drugs but develops resistance within months. The study found that MET gene amplification drives this resistance, and a blood test can detect its presence prior to relapse.
Glybera, first gene therapy drug approved in the Western world, was developed after a decade-long search for its genetic mutation. The European Medicines Agency granted marketing approval on November 2, 2012, after several rejections and appeals. Glybera's efficacy was questionable, but its safety was not an issue.
Researchers have developed a new gene delivery method using magnetically targeted nanoparticles that can effectively deliver genes to injured arteries without causing side effects. The technique, which uses stents as a platform for magnetic guidance, shows promise in overcoming current limitations of gene therapy vectors.
Recent progress toward developing effective gene therapies for regenerative surgery has shown promising results in growing skin, bone, and other tissues. However, translating experimental gene therapy methods into clinical trials remains a difficult challenge due to technical and cost-effective issues.
A study published in JAMA found that a specific genetic variation is associated with improved survival in some cases of pulmonary fibrosis. The researchers identified the MUC5B promoter polymorphism as a key factor, which may lead to the development of new treatments for this deadly lung disease.
The University of Maryland Medical Center is now offering genetic testing to help doctors determine the best medication for each patient, based on their unique genetic makeup. This personalized medicine initiative aims to improve the quality of care provided to cardiac patients.
Researchers at Massachusetts General Hospital have identified a gene variant that predicts weight loss after gastric bypass surgery. The variant, located on chromosome 15, is associated with an average weight loss of nearly 40% in individuals who carry two copies of the beneficial version.
Researchers analyzed 1,647 brain tissue samples to identify molecular networks disrupted in Alzheimer's disease, highlighting the crucial role of TYROBP in immune system dysfunction. This study provides a new framework for understanding Alzheimer's mechanisms and developing potential therapies.
Researchers have linked 17 genes to juvenile idiopathic arthritis, a significant increase from previous findings of three genes. The study analyzed over 2,800 patient DNA samples and provides new insights into the disease process.
The Hospital for Special Surgery will establish a genomics center to study rheumatoid arthritis and systemic lupus erythematosus using genomic approaches, aiming to develop more effective therapies. The center will focus on epigenetic therapy and personalized medicine, analyzing gene expression and environmental influences.
A new treatment for inherited ALS has passed an early clinical trial with no serious side effects, successfully introducing the drug into the central nervous system. The phase 1 trial suggests the approach may be effective against other mutated genes causing central nervous system disorders.
Scientists have discovered 14 new genes associated with childhood arthritis, which could lead to more effective treatments and improved outcomes. The study's findings may also enable clinicians to identify children who require specific therapy earlier, ultimately reducing the impact of the disease on their quality of life.
A new genetic screen has identified the MKK4 gene as a promising therapeutic target to enhance liver regeneration. The study reveals that inhibiting MKK4 can increase hepatocyte production and survival, leading to healthier livers and improved long-term survival in mice.
Researchers from BUSM have developed a genetic signature for COPD from airway cells, providing a new way to study the disease and monitor patient response to treatments. This approach has shown consistent gene-expression changes in both airway and lung tissue cells in individuals with COPD.
Researchers have developed nanoparticles that improve siRNA delivery, enabling more efficient silencing of specific genes. The approach shows promise in addressing the challenge of targeted delivery for gene therapies.
A study at Northwestern University and Dartmouth's Geisel School of Medicine found that patients with improved responses to mycophenolate mofetile (MMF) therapy share a distinct gene expression pattern in skin. This signature can potentially guide targeted treatment for patients with scleroderma.