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.
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.
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.
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.
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.
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.
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.
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.
A Canadian pilot study on gene therapy for Fabry disease shows the treatment is working and safe, enabling patients to produce normal levels of the defective enzyme. The trial, led by Dr. Aneal Khan, treated five men with a single dose of gene therapy, which resulted in stable patients who are no longer requiring replacement therapy.
Researchers found that genes affecting cilia function are linked to diabetes, kidney failure, and liver fibrosis in both rare genetic disorders and the general public. The discovery opens up new possibilities for targeted treatments and gene therapies.
Researchers identified key risk genes associated with schizophrenia and their impact on antipsychotic medication efficacy. Examining these single nucleotide polymorphisms (SNPs) can help differentiate between responsive and treatment-resistant patients.
Researchers have developed an immune tolerance platform called ImmTORTM to overcome challenges in gene therapy, including immunogenicity and durability. The addition of ImmTOR nanoparticles to AAV vectors has shown potential to enhance efficacy, safety, and durability by mediating more efficient transgene expression.
Researchers have developed improved gene vectors for ocular gene therapy, allowing for widespread delivery and reducing risks associated with traditional approaches. These vectors are being tested in clinical trials and have shown promise in restoring daylight vision in animal models of achromatopsia.
A new biomarker has been identified that can determine individual treatment duration for tuberculosis patients, reducing the need for prolonged therapy. The biomarker is based on an RNA signature from 22 genes and was developed using patient cohorts from Germany and Romania.
Researchers develop DNA modifying epigenetic therapy to transform immune killer T-cells into "super soldiers" with enhanced ability to kill cancer cells. The therapy uses available chemotherapy drugs to remove epigenetic tags, turning key genes back on and turbocharging the cells' killing function.
Researchers from Indiana University have identified key genetic changes in the interstitial kidney tissue of people with diabetes, which could lead to a revolutionary new genetic approach to treating kidney disease. The study found that important genes change when a scar forms on the interstitium, a previously undercharacterized part o...
Researchers found higher than expected genetic changes in a group of 100 MND patients, suggesting routine genetic testing may be beneficial. The study recommends genetic testing for all MND patients, regardless of family history, to improve disease subclassification and tailored treatments.
Researchers at Wyss Institute and Google Research used machine learning to design highly diverse AAV capsid variants that can evade neutralizing antibodies. The approach produced over 57,000 variants with improved functional diversity, potentially leading to improved gene therapies and reduced immunogenicity.
Researchers used artificial intelligence to generate a large library of distinct AAV capsid variants, achieving a 60% viable yield. This approach overcomes the limitation of current vectors and expands the number of diseases treatable with gene therapies.
A new AAV-delivered gene therapy strategy uses immunomodulation to reduce inflammation and improve efficacy. By incorporating TLR9-inhibitory sequences into the AAV genome, researchers have enhanced expression of transgenes in mice, suggesting potential for higher efficacy.
Researchers have developed targeted ultrasound techniques to treat various brain diseases such as Alzheimer's, Parkinson's, and stroke. The new methods aim to improve brain functions by activating functional neurons without significant side effects.
The Canadian Neuromuscular Disease Registry has facilitated over 125 research projects and secured funding for patients across Canada. With data from over 4,000 patients, the registry provides valuable insights into neuromuscular disease outcomes and care.
Researchers discovered that three patients with DOCK8 deficiency spontaneously repaired their faulty genes through somatic reversion, restoring normal immune function. This breakthrough has implications for future therapies and treatments for the often-fatal disease.
The AAVCOVID vaccine program has received a $2.1 million grant to support phase I clinical trials overseas, with single-dose vaccines that can be stored at room temperature for up to one month. This stability could enable distribution in regions with limited infrastructure.
Researchers at Children's Hospital of Philadelphia developed a new gene therapy vector that produces more hemoglobin with a lower dose, minimizing toxic side effects. The vector, ALS20, was found to be significantly more effective than current vectors in treating beta-globinopathies.
Scientists at KAUST developed rules to protect inserted genes from nematode natural defenses, allowing for multiple generations of gene expression. A web application helps analyze DNA sequences for PATC watermarks, facilitating research on transgene silencing resistance.
Researchers developed a gene therapy strategy to treat Leber congenital amaurosis by adding copies of the normal CRX gene under its native control mechanism. This approach restored some CRX protein function and drove expression of opsins in patient-derived retinal organoids.
Researchers at Mayo Clinic have developed the first hybrid gene therapy for treating long QT syndrome, a genetic heart rhythm condition. The therapy targets the KCNQ1 gene and has shown potential therapeutic efficacy in two in vitro model systems using beating heart cells reengineered from patient blood samples.
Researchers have found a new type of genetic change in people with hypertrophic cardiomyopathy (HCM), which can cause sudden death. This discovery will help doctors predict which family members need to be monitored and which can be ruled out from further tests.
A phase 2 clinical trial using a personalized treatment approach found increased survival rates among patients with metastatic tumors in gastroesophageal cancers. The study used genetic profiling to tailor treatments, resulting in a 66% one-year survival rate and a median survival time of 15.7 months.
The study investigates the biodistribution of AAV gene transfer vectors in nonhuman primates using quantitative positron emission tomography (PET). The results show that both AAVrsh.10 and AAV9 vectors distribute primarily to the liver, with lesser detection in the brain after intravenous administration.
A team of researchers from Ruhr-University Bochum has developed a novel approach to treat spinal cord injuries by stimulating nerve cell regeneration with a designer cytokine. In a groundbreaking study, they successfully restored walking ability in paralyzed mice, paving the way for future human trials.
Researchers at Massachusetts General Hospital have developed a gene therapy strategy that effectively treats mice with a mutated TSC2 gene, causing the growth of noncancerous tumors. The treatment extends survival to 462 days and reduces brain damage in mice, suggesting potential for human clinical trials.
Researchers found that the HTT gene mutation affects brain and body growth, leading to increased susceptibility to brain cell death, even in children as young as six. Gene therapy trials are underway to slow disease progression and potentially prevent onset by delivering gene therapy to carriers.
Antisense oligonucleotides (ASOs) target disease-causing proteins, but can affect non-targeted proteins causing side effects. Researchers have developed a DNA/DNA double-stranded oligonucleotide that enhances ASO efficacy and stability in the body.
Researchers found that decreased activation of gene LEF1 disrupts neuronal function and promotes hyperexcitability in brain cells, a hallmark of bipolar disorder. Increasing LEF1 expression may lead to new drug targets and biomarkers for lithium nonresponsiveness.
Scientists at Tel Aviv University have developed a new gene therapy that replaces the genetic defect causing deafness in mice. The treatment enabled cells to continue functioning normally, preventing gradual deterioration of hearing.
Researchers have discovered a new way to deliver DNA-based therapies for diseases using polymers, which could lead to more affordable gene therapies or vaccines. The polymer technology works by packaging the nucleic acids in a carrier that protects them from degradation, allowing them to reach their target cells.
Researchers at the University of Exeter have identified a new treatment approach for mitochondrial diseases, such as Leigh Syndrome, by using novel drugs that metabolically reprogram mitochondria to generate energy. The study successfully normalized or improved energy production in genetically mutated microscopic worms.
Researchers Gustavo D. Aguirre, Jean Bennett, and Albert M. Maguire receive $1 million prize for developing FDA-approved gene therapy for Leber congenital amaurosis, a genetic disease causing visual impairments. Their work has enabled routine treatment of the condition, restoring vision in children and adults.
A new study identified an adenovirus gene therapy vector carrying a VEGF isoform that can improve uterine blood flow in placental insufficiency. Reduced uterine blood flow and lack of bioavailable VEGF are major causes of severe fetal growth restriction, leading to serious neonatal morbidity and death.
Researchers have discovered AAV capsid-promoter interactions in the non-human primate brain, which dictate cell-specific transgene expression. This finding has profound implications for vector design in gene therapy, challenging the traditional concept of the AAV capsid as a delivery truck.