Targeting therapeutic genes to the lungs offers potential for managing serious lung diseases that do not respond to other treatments. Preclinical and human clinical studies have targeted various genetic diseases, including cystic fibrosis, asthma, and lung cancer.
Researchers successfully used CRISPR-Cas9 technology to repair genetic mutations in blood stem cells of patients with chronic granulomatous disease, a rare and life-threatening disorder. The engineered cells maintained their gene edits long-term without side effects when implanted into mice.
Researchers successfully repaired a defective gene in blood-forming stem cells from patients with X-linked chronic granulomatous disease, suggesting a potential treatment approach. The study used CRISPR-Cas9 technology to correct a specific mutation in the CYBB gene, restoring normal functioning of white blood cells.
A new research from Michigan Medicine supports combining immune checkpoint blockade with gene therapy to fight gliomas. The approach showed stronger results when used in combination with either depletion of immunosuppressive cells or immune checkpoint blockade.
Katherine High discusses gene therapy progress for hemophilia and inherited retinal disorders, highlighting key scientific, regulatory, and commercial challenges. Her leadership in the field has been critical to its current success.
Gene therapy for liver disease is advancing with the help of adeno-associated viral (AAV) vectors, offering new therapeutic possibilities. The article discusses preclinical and clinical studies that have led to a better understanding of immune responses to AAV gene therapy.
Researchers evaluated two adeno-associated virus vectors in non-human primates to assess gene expression efficiency and immunogenicity. The results provide insight into the safety and effectiveness of these gene therapy approaches before initiating clinical trials in humans.
A new study published in Nature provides key insights into mitochondrial replacement therapy, a gene-therapy technique that aims to prevent the transmission of harmful mitochondrial gene mutations from mothers to their children. By selecting egg donors with compatible haplotypes, clinicians may achieve higher success rates for producin...
Researchers at the University of Pennsylvania have developed a dual gene therapy approach using CRISPR/Cas9 to deliver key components to treat hemophilia B. The study showed stable Factor IX activity in newborn and adult mice, with all treated mice surviving a partial liver removal without complications.
A novel mapping technique combines imaging and electroanatomical mapping to guide gene therapy injections into viable myocardium with reduced contractile ability, increasing blood flow in patients with refractory angina. This approach shows promise for repairing damaged heart muscle.
A study of over 500,000 patients found that high-intensity statin therapy significantly lowers risk of death from atherosclerotic cardiovascular disease. The study suggests optimizing intensity of statin therapy for improved secondary prevention.
Researchers at Stanford University School of Medicine have successfully repaired the gene causing sickle cell disease using CRISPR gene editing technology. The corrected human hematopoietic stem cells were then transplanted into mice and showed promise in producing functioning hemoglobin molecules.
A WSU researcher has developed a modified viral vector that reduces the risk of cancer cells and can be used for multiple blood diseases. The new delivery system is being translated into a stem cell gene therapy to treat a life-threatening immunodeficiency in newborns.
A study has discovered a crucial part of the genetic machinery that switches on the Neuroglobin gene, which is associated with protecting the brain and reducing severity of damage due to stroke and Alzheimer's disease. The research opens up new opportunities for treating AD using gene therapy.
Researchers at the University of Helsinki discovered that gene therapy stimulating blood vessel growth in the heart can alleviate cardiac atrophy caused by doxorubicin. The study found that this treatment prevented blood vessel rarefaction in mouse hearts, providing a potential solution for protecting patients from cardiotoxicity.
The Cell Press has partnered with The American Society of Gene and Cell Therapy (ASGCT) to publish its four journals, including Molecular Therapy, starting January 2017. This partnership aims to improve the dissemination of research breakthroughs in gene and cell therapies.
Researchers have successfully used gene therapy to correct the faulty NPC1 gene in mice with Niemann-Pick disease type C1, improving symptoms and lifespan. The treatment has the potential to halt disease progression and improve quality of life for patients.
A portable device enables medical staff to genetically modify blood stem cells at a lower cost and with reduced staffing requirements. The technology has the potential to make gene therapy more accessible to patients in developing countries, reducing the need for expensive clean rooms.
Researchers have prevented the development of Alzheimer's disease in mice by using a virus to deliver a specific gene, PGC-1 - alpha, into the brain. The study opens avenues for potential new treatments for the disease and suggests that injections of the gene may be beneficial in the early stages of the disease.
Researchers developed a lentiviral vector-based gene therapy approach to deliver normal copies of the alpha-iduronidase gene to HSCs, indicating safety and efficacy in mice. The study sets the stage for a clinical trial to determine if MPS I patients can be successfully treated with this approach.
Researchers developed a precision-engineered gene therapy virus that selectively silences BCL11A, reducing signs of sickle cell disease and increasing fetal hemoglobin production. The approach has the potential to substantially increase the ratio of non-sickling versus sickling hemoglobin.
Researchers found that naturally infected chimpanzees developed antibodies able to neutralize multiple AAV serotypes, a hurdle for gene therapy. The study provides insights into the immune response to natural AAV infection and its implications for gene delivery strategies.
Researchers developed a hydrogel patch that delivers gene therapy, chemotherapy, and thermal ablation to treat colon cancer. The patch achieves complete tumor remission in non-resected tumors and prevents recurrence when applied after surgery.
Researchers from the University of Pennsylvania have shown that early exposure to the human IDUA protein increased immune tolerance, allowing for widespread expression of the enzyme and resolution of brain lesions. The study informs the planning of first-in-human trials for MPS I treatment.
A new gene therapy approach successfully delivered a therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival. The treatment holds promise for developing similar therapies for humans and targeting additional brain disorders.
A new study found no serious adverse events related to gene therapy in patients with advanced Parkinson's disease five years after treatment. The long-term safety profile of the therapy is promising, lending support to its potential as a treatment for this debilitating disease.
Researchers describe expanding scope of gene therapy targeting CNS diseases, including Alzheimer's and ALS. The article highlights rapid progress in viral vector development and delivery strategies for treating these conditions.
A new study discovered 172 novel cancer gene-drug combinations that selectively kill cancer cells without harming healthy cells. This breakthrough expands the number of actionable mutations and provides a promising approach to personalized cancer therapy.
Researchers have developed a lung-targeted gene therapy strategy that may improve treatments for inherited diseases like emphysema. The new approach, which delivers a normal copy of the gene to lung cells, shows promise in reducing disease severity and increasing protein production.
Researchers outline recent progress in clinical applications of retinal gene replacement therapy and preclinical advances in gene-specific therapy for photoreceptor diseases. Gene therapy strategies are being developed to treat a broader range of disorders affecting vision, providing new hope for individuals with eye diseases.
A recent study by researchers at the University of Georgia found that silencing of the OXT gene may impact social behaviors, including recognizing emotional states of others. Participants with higher methylation levels of the OXT gene showed difficulty in describing facial emotions and had increased anxiety about relationships.
A commentary suggests that gene therapy treatments for rare diseases should be valued based on long-term cost savings. The authors propose tying costs to efficacy and creating a federal initiative to support new therapies.
Researchers found that a tumor suppressor gene helps repair UV damage in cells, reducing the risk of skin cancers like melanoma. The study suggests this gene may serve as a biomarker for skin cancer prevention and offers hope for new drug targets.
A new gene therapy has been developed to repair lungs damaged by chronic allergic asthma, reducing inflammation and improving airway structure and function. The study used a novel biodegradable platform to deliver the thymulin gene therapy, showing effective results in mice with experimental asthma.
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...
Promising results from clinical trials of globin gene transfer have eliminated the need for blood transfusions in some individuals with beta-thalassemias. Gene editing technologies hold promise to correct beta-globin deficiencies and reactivate fetal hemoglobin production, potentially leading to a cure for severe globin disorders.
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.
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 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.
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.
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.
Cell therapy is an emerging medical science focusing on innovative therapeutic approaches to treat blood diseases and infections. The EBMT Cellular Therapy and Immunobiology Working Party aims to foster cellular therapy in Europe by promoting exchanges, cooperation, and the development of new technologies.
Three researchers have received ACGT Young Investigator Grants to develop new treatments for leukemia/lymphoma, melanoma, and blood cancers. Dr. Yvonne Chen aims to improve CAR-T therapy, while Dr. Brent Hanks seeks to block the immune system's ability to destroy cancers using immunotherapy.
Researchers at UVA University have successfully used a synthetic gene to manipulate the behavior of mice and zebrafish by applying magnetic fields. The breakthrough could lead to new treatments for neurological diseases such as schizophrenia and Parkinson's disease.
Researchers found that deleting the huntingtin gene in adult mice does not lead to lethal consequences, offering hope for treatment strategies involving gene silencing. The study suggests that gene suppression or editing strategies may be safe for adults, but further research is needed to understand the long-term effects.
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.
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.
Recent gene therapy developments focus on viral vectors like AAV for safe gene transfer, and new gene editing tools offer targeted gene inactivation or insertion. These approaches aim to treat multiple diseases with platform strategies.
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.
Researchers have successfully treated a genetic disorder using a viral vector to deliver genome-editing components, correcting the disease-causing mutation. The treatment improved survival in newborn mice but showed poor results in adult animals, highlighting the need for further adjustments to the gene-editing system.
Researchers have successfully corrected a blindness-causing gene mutation in stem cells derived from a patient's skin cells, offering hope for personalized, precision medicine. The technology uses CRISPR/Cas9 gene editing to repair the damaged gene, and the corrected tissue can be transplanted without harm.
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%.
Researchers successfully tested gene therapy combined with low-dose chemotherapy in five patients aged 7-24 with worsening immune systems due to SCID-X1. The therapy showed substantial improvements in immunity and clinical status, particularly in the first two patients who received treatment.
Researchers have made encouraging results using gene therapy to treat cystic fibrosis by inserting a healthy copy of the CFTR gene into affected cells. The approach restored chloride and fluid transport in both mouse models and human-derived cell cultures, suggesting a potential cure for the genetic disorder.
Researchers successfully treated dogs with Duchenne Muscular Dystrophy using gene therapy, developing a miniature version of the dystrophin protein that protects all muscles. The treatment uses a common virus to deliver the gene, which has shown no symptoms in human bodies and is being planned for human clinical trials.
Biotech expert Mitchell Finer shares his experiences and strategies for achieving commercial success, including the importance of clinical data and access to capital. He also discusses the growth of companies like bluebird bio and Cell Genesys, and the potential for gene therapy to revolutionize treatment options.
A phase III clinical trial's results may lead to the first approved gene therapy product in the US for treating Leber congenital amaurosis type 2 (LCA2). The treatment, SPK-RPE65, could improve patients' ability to see and function at night or in dimmer light levels.
A study funded by the NIH has shown that gene therapy can preserve vision in dogs with late-stage retinitis pigmentosa. The treatment halted disease-associated cell death and preserved photoreceptor structure, improving visual performance under dim light conditions. Further research is needed to assess its safety and potential toxicity...