Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Using two therapies together has been shown to effectively restore vision in dogs with inherited blindness. The treatment approach may have implications for human patients as well. Researchers believe that combining gene therapy and selective photoreceptor cell destruction could lead to better therapeutic outcomes.
Researchers at the University of Colorado Cancer Center have developed a new technology that uses cholesterol rafts to deliver genetic payloads into cancer cells. This innovative approach overcomes the long-standing challenge of delivering nucleic acids across cell membranes.
A special issue of Translational Research highlights the progress and challenges of gene therapy research, focusing on clinically meaningful studies that combine patient observations with smart experiments. The approach of gene therapy may be applicable to all genetic disorders, offering a promising path for treatment and cure.
Researchers develop a gene therapy technique to increase enkephalin, an opioid compound, in the bladder wall, reducing pain signals. The treatment approach shows promise for severe chronic pain conditions like bladder pain syndrome/interstitial cystitis.
Researchers at Vall d'Hebron Institute of Oncology have successfully eliminated mouse lung tumors by inhibiting the Myc protein, a key regulator of gene expression and cell growth. The study confirms that Myc-targeted therapy is a safe and effective option for treating various types of cancer, with no signs of resistance or side effects.
After over 20 years, a team led by Professor Merlin Crossley has found the genetic puzzle piece for hemophilia B Leyden, a rare disorder that improves symptoms after puberty. The breakthrough demonstrates the importance of persistence and curiosity in scientific research.
A new US patent application allows for methods of restoring visual responses using optogenetic compounds, covering channelrhodopsin and halorhodopsin variants. The approved patent will substantively expand RetroSense's IP estate, providing broad protection for their gene therapies.
Researchers have identified seven new genetic regions associated with AMD, explaining up to 65% of the genetics of the disease. The study combined existing data from over 17,000 patients and 60,000 people without AMD, revealing genes involved in immune system signaling, lipid metabolism, and blood vessel development.
Researchers develop lipid nanoparticles as vectors in gene therapy to deliver therapeutic genes to cells without degrading within the organism. The technology improves drug absorption for insoluble or poorly soluble molecules, offering a promising alternative for diseases with no effective treatment.
A study published in Nature describes how the CPEB1 protein 'takes the brakes off' the production of proteins associated with cell proliferation and tumor progression. The mechanism, discovered using Hodgkin lymphoma cells, has been proposed as a general regulatory system that enhances cancer spread.
A new gene therapy trial aims to promote the regeneration of heart tissue by encouraging the body to deploy more stem cells to the injury site. The therapy, called JVS-100, has shown clinically significant improvements in exercise levels in patients with ischemic heart failure.
A novel drug delivery strategy has been shown to neutralize mutant RNA toxicity and eliminate myotonia symptoms in mice with myotonic dystrophy. The approach uses antisense oligonucleotides and peptide-linked morpholino oligonucleotides to target the disease-causing mutation.
Scientists have discovered a crucial role of ADAMTS7 enzyme in building up cells in coronary arteries, contributing to the development of coronary heart disease. The study found that a specific gene variant reduces risk by 50% and slows down plaque growth.
Researchers at Case Western Reserve University discovered a missing RNA element that significantly improves the effectiveness of gene therapy. The element, called Genomic RNA Packaging Enhancer element (GRPE), coordinates the production and filling of genetic material in viral vectors.
Aravind Asokan, assistant professor of genetics at UNC School of Medicine, received the award for his significant contributions to gene therapy. His lab developed a synthetic viral toolkit to unravel viral infectious pathways and produce novel vectors for gene therapy.
Researchers have discovered a vast potential for noncoding RNAs to be used as therapeutic and diagnostic tools. Long noncoding RNAs play significant roles in various diseases, including cancer, diabetes, and other conditions. The emerging concept of microRNAs functioning as hormones has also led to new avenues for disease treatment.
A landmark clinical trial published in Ophthalmology found that genetic variants do not predict treatment response to Lucentis and Avastin for wet AMD. The study's findings clarify the value of genetic testing for this serious eye disease, contradicting previous hopes for personalized medicine.
Researchers from UAB have shown that gene therapy can cure type 1 diabetes in large animals with a single session. The therapy uses a 'glucose sensor' to regulate blood sugar levels, reducing diabetic hyperglycemia and preventing complications.
Researchers developed a system to artificially simulate natural complex interactions between proteins that regulate genes, allowing for precise control over gene activation. This breakthrough enables scientists to better understand human gene regulation and develop new gene-based therapies.
The EU has announced €38 million funding for research into rare diseases, aiming to develop new diagnostics and treatments through global data sharing. Advances in DNA sequencing have brought personalized treatments closer, but scientists now need to collate data to identify genetic causes of diseases.
A novel aptamer has been developed that specifically targets and stimulates human immune cells, significantly increasing the effectiveness of immunotherapeutic drugs. The aptamer enhances the ability of activated T cells to proliferate and produce immunostimulatory cytokines.
A new study from the University of Pennsylvania School of Medicine found that gene therapy improves vision in patients with Leber congenital amaurosis, but also advances retinal cell degeneration. The research provides a more nuanced understanding of the disease and its treatment.
Researchers have successfully treated Duchenne muscular dystrophy in dogs using gene therapy, reducing inflammation and improving muscle strength. The study, published in Molecular Therapy, provides a quantum leap forward in fighting this devastating disease, paving the way for future human clinical trials.
Researchers developed a new gene therapy approach called exon skipping, which bypasses disease-causing mutations in genes to restore normal expression and protein production. This strategy has shown promising results in treating muscular dystrophy, with improved muscle force and increased body weight and muscle mass.
A new gene has been identified with a key role in obesity and diabetes, regulating fat storage and energy expenditure. The study suggests that blocking this gene could lead to therapies for obesity and related illnesses.
Researchers have developed a new method for precisely altering the genomes of living cells, enabling targeted gene insertion and deletion with increased accuracy. This breakthrough technology has far-reaching potential applications in biofuel production, disease research, and therapy development.
Scientists have identified a rare form of active 'jumping genes' in mammals, specifically in bats. This discovery opens up new avenues for studying evolution and developing tools for gene therapy.
Eczema is a persistent skin rash that can be triggered by inadequate Ctip2 protein, leading to inflammation and immune dysfunction. Researchers have identified two ways in which improper Ctip2 function contributes to eczema, paving the way for personalized treatments and new therapies.
A 10-year follow-up study shows promising outcomes for patients who received gene therapy to rebuild their blood vessels. The five- and 10-year survival rates were comparable to those of patients treated with traditional medical therapy, with some cases showing improved outcomes.
Two novel treatments for retinitis pigmentosa have shown promising results in mouse models, with stem cell grafts improving vision and gene therapy restoring photoreceptor cells. The treatments aim to provide a potentially unlimited supply of cells and offer an alternative to immunosuppression.
A clinical trial of a gene therapy cocktail has shown promising results in treating Canavan disease, a rare and fatal neurodegenerative disorder. The treatment uses an adeno-associated virus to deliver a replacement ASPA gene to the brain, reducing NAA levels and improving symptoms.
Cedars-Sinai researchers have successfully converted ordinary heart cells into pacemaker cells using a single gene, Tbx18. The new cells generate electrical impulses and are indistinguishable from native pacemaker cells, offering a potential alternative to electronic pacing devices.
Five young investigators from the Asia-Pacific region were awarded prestigious grants for their top-ranking abstracts. The IOF Young Investigator Award valued at USD 1,000 and invited them to present their work in oral presentations during a special plenary session.
Scientists are harnessing big data analytics and genomics to tailor fertility treatments to individual women. Dr. Piraye Yurttas Beim's work at Celmatix Inc. aims to develop non-invasive diagnostic tests to identify genetic drivers of infertility, enabling personalized recommendations for effective treatments.
Researchers discovered that genetic mutations are only one piece of the puzzle, and biological factors and cell behavior contribute to tumour growth and therapy failure. The team found that some cancer cells were responsible for keeping cancer growing, while others were transient and stopped within days.
Researchers successfully repair and read-through stop mutations responsible for Usher syndrome, offering hope for patients with the disease. The team developed gene-based treatment strategies using zinc-finger nuclease technique and aminoglycosides, which show promise in preclinical studies.
Researchers at Case Western Reserve University School of Medicine identified a genetic factor KLF4 that prevents blockages from forming in blood vessels. The study found that a shortage of KLF4 makes the lining more prone to plaque buildup and clot formation, while sufficient levels protect against toxins and disease.
Researchers at the University of Manchester have identified 14 new genes linked to rheumatoid arthritis, shedding light on why the condition disproportionately affects women. The study's findings could lead to personalized treatment approaches and improved disease management.
Researchers explored the benefits of adding radionuclide therapy to cancer treatment regimens for patients with advanced disease. Studies found improved tumor control rates and longer patient survival without significant side effects, suggesting a potential paradigm shift in cancer control.
The study found that resveratrol induces cell death in cancer cells with normal p53 expression, but not those with defective p53. Introducing a normal copy of p53 into p53-defective cancer cell lines makes them sensitive to resveratrol's anti-tumor effects.
Dr. Richard Casaburi leads research on COPD, a disease affecting millions, to identify genetic factors and develop new treatments. The study, funded by NIH, aims to understand what genetic deficiencies contribute to COPD development and progression.
Researchers identified 70 genetic mutations in Burkitt lymphoma tumors, including a new mutation that accelerates tumor growth. The findings could lead to the development of new treatments or targeted therapies for cancer patients.
Researchers at Moffitt Cancer Center have found that men with prostate cancer who receive androgen deprivation therapy are susceptible to heightened fatigue if they carry specific variants of the IL6 and TNFA genes. This discovery highlights the importance of personalized medicine in tailoring therapies to a patient's genetic profile.
Researchers have developed targeted T-cell therapies that can recognize and destroy glioma stem cells, which are resistant to traditional treatments. The new approaches involve genetically modifying T-cells to target specific antigens on tumor cells, potentially improving treatment efficacy and reducing cancer recurrence.
Researchers at OHSU have successfully demonstrated a new gene therapy method that can replace mutated mitochondrial DNA with healthy copies in human cells. This breakthrough has the potential to prevent devastating diseases passed from mother to infant.
Researchers at Johns Hopkins have identified a neuroprotective drug called ethoxyquin that effectively prevented nerve damage in mice treated with chemotherapy drugs, as well as protected nerves in diabetic rats and the mouse model of HIV. This finding has significant implications for developing new treatments for neuropathy.
Researchers from Johns Hopkins and Northwestern universities discovered how to control the shape of DNA nanoparticles that move through the body. The shapes of these carriers may make a big difference in treating cancer and other diseases. Using computer models, they found that worm-shaped particles resulted in 1,600 times more gene ex...
Researchers at Thomas Jefferson University have found that low levels of the protein S100A1 are associated with critical lower limb ischemia, a condition characterized by reduced blood flow to legs. The study suggests that S100A1 could be a potential therapeutic target in critical limb ischemia to promote revascularization.
A research team at New York University has determined how cells that cause inflammatory ailments differentiate from stem cells. The study found hundreds of new genes involved in the function and development of these cells, which can be used to design new therapies for diseases such as Crohn's disease, multiple sclerosis, and arthritis.
A team of researchers identified genetic causes of the most common form of breast cancer, Estrogen-Receptor positive Luminal A subtype, providing clues for new therapeutic targets. They also found a molecular similarity between basal-like breast cancers and high-grade serous ovarian tumors.
New research found that lithium-responsive patients exhibit increased expression of anti-apoptotic genes like Bcl2 and IRS2, while those who don't respond show decreased Bcl2 levels. This study provides insight into personalized treatment for bipolar disorder.
The study reveals that human brains are more similar than different, with the same basic molecular blueprint across individuals. Key findings include regional similarity in biochemistry, homogeneous cortex structure, and widespread gene expression across brain areas.
Researchers develop refined gene therapy approach that safely restores the immune system of children with ADA-deficient SCID. Chemotherapy conditioning regimen prior to treatment significantly increases ADA enzyme levels and partial immune reconstitution in patients.
Researchers at UCLA have developed a gene therapy regimen that safely restores immune systems to children with ADA-deficient severe combined immunodeficiency (SCID), a devastating disease. The treatment showed promising results in restoring immune function to three out of six patients, offering new hope for these children.
Researchers have successfully restored the immune systems of some children with SCID using a refined gene therapy approach, eliminating the need for life-long injections. The treatment involves inserting a healthy copy of the ADA gene into bone marrow stem cells, preventing immune-destroying toxicity.
Researchers highlight RNA's self-assembling properties, but also discuss challenges such as stability and targeting in therapeutic applications. The article provides a detailed review of the field, discussing its potential for treating various diseases.
Researchers restored olfactory function in mice with congenital anosmia by introducing a healthy copy of the IFT88 gene, leading to improved feeding behavior and weight gain. The study suggests a potential therapeutic approach for treating people born with anosmia.
Scientists have restored the sense of smell in mice through gene therapy, providing a hopeful sign for those born without or losing their sense of smell due to disease. The breakthrough may also aid research into other cilia-related diseases, such as polycystic kidney disease and retinitis pigmentosa.
Studies reveal paradoxical roles of PML in regulating fat-burning metabolism and its impact on breast cancer cells, hematopoetic stem cell maintenance, and obesity. Enhanced cellular metabolism provides energy to breast cancer cells, while inhibition of fatty acid oxidation poses a risk to replenishment of HSCs.
Researchers have discovered a molecule that could lead to a future ALS treatment, modifying the course of the disease in zebrafish models. The EphA4 receptor was found to play a key role in the mechanism of ALS, with its expression linked to disease severity and recovery.