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 $14.6 million NIH grant will fund an international, multidisciplinary effort to leverage recent genetic discoveries into possible treatments for AMD. The investigation aims to explore the role of complement factor H and factor B genes in the immune system and develop diagnostic tools and treatments.
Researchers at St. Jude Children's Research Hospital developed a mouse model that explains why gene therapy treatment caused leukemia in some severe immune deficiency patients with XSCID. The study found that the disease itself makes mice susceptible to cancer caused by gene therapy, offering hope for safe treatment.
A recent study has found that thrombotic complications occur in 5.2% of children with leukaemia, with the highest risk during antileukaemic therapy induction phase. The researchers have identified several factors associated with an increased risk of thrombosis and explored the role of genetic determinants.
Queen's University researchers found that two genes cooperate to protect aortic smooth muscle cells from death, pointing to new therapies for preventing heart attacks and strokes. The study's findings suggest that delivering the HO-1 gene using gene therapy may help stabilize plaques and prevent clogging in arteries.
Researchers used gene therapy to deliver glial-derived neurotrophic factor (GDNF) directly to the brain cells of mice with Huntington's disease, protecting neurons from degeneration. The study showed improved behavioral function and reduced symptoms in mice treated with GDNF, suggesting a new approach to forestall disease progression.
Researchers used gene therapy to treat ovarian cancer in a mouse model, achieving complete inhibition of tumor growth in some mice and significant inhibition in others. The treatment offers a promising alternative to harsh current treatments for this deadly disease.
Researchers developed gene therapy using manganese superoxide dismutase to protect mice from radiation damage. The treatment showed no changes in body weight, little bone-marrow damage, and longer lifespan in mice exposed to varying doses of whole-body radiation.
Researchers used adeno-associated virus to insert genes for interleukin-4 (IL-4) or interleukin-10 (IL-10) into insulin-producing beta cells, preventing hyperglycemia in non-obese diabetic mice. Gene therapy shows promise as a viable method for preventing type 1 diabetes in genetically at-risk individuals.
A novel therapeutic approach has been discovered by the University of Manchester team, using a DNA-binding protein to stimulate genes and promote nerve growth. The preclinical results have been encouraging, with initial-stage clinical trials in the US showing promising progress in managing diabetic neuropathy.
Researchers at Stanford University School of Medicine have made significant progress in developing RNAi gene therapy, a promising approach to treating organ-wide diseases. However, they also encountered unexpected side effects, including liver toxicity in mice, which hindered their progress. Despite this setback, the team was able to o...
Researchers have successfully treated a chicken model of LCA1, a form of childhood blindness, using gene therapy. Six out of seven treated chicks developed sight, despite only a small percentage of receptor cells being infected by the virus. This study demonstrates the potential for gene therapy to treat this form of blindness in humans.
Researchers at the University of Florida have successfully restored vision to chickens with a genetic defect causing blindeness. Five out of seven treated chickens displayed near-normal visual behavior and responded to light, offering hope for an eventual vision-restoring therapy for children with inherited childhood blindness.
In-vivo gene therapy has successfully restored immune function in three out of four dogs treated for XSCID. The treatment approach eliminates the need for ex-vivo cell manipulation and culture, potentially reducing adverse side effects and improving long-term efficacy.
Researchers have developed a drug that increases SMN protein levels in SMA patients, improving their quality of life. Another study shows promise for treating X-linked adrenoleukodystrophy (X-ALD) and Duchenne muscular dystrophy (DMD) with valproate and anti-sense molecules.
A new study by Harvard Medical School researchers found that visual stimulus turns up the expression of some genes and turns down others, shaping the brain. The study identified distinct sets of genes that respond to visual input at different ages, suggesting a more holistic view of gene function in neural development.
A recent mouse study led by the Salk Institute has discovered that healthy copies of the IL2RG gene used in X-linked severe combined immune deficiency (X-SCID) gene therapy can promote cancer development. One-third of mice treated with the gene developed lymphoma later in life.
Researchers have made progress toward a new form of light-activated gene therapy for cartilage repair, which is safe and compatible with existing surgical techniques. The therapy uses UV light to target specific cells and promote tissue growth, offering hope for treating knee injuries and potentially other conditions.
The landmark book celebrates the centennial of Alzheimer's discovery, narrating its evolution from a major cause of late-life dementia. The publication brings to life classic studies defining the disease research, featuring contributions from prominent researchers.
The study reveals high molecular heterogeneity in multiple myeloma, with new recurrent amplifications and deletions pointing to yet-to-be-discovered oncogenes and tumor suppressor genes. These findings define new disease subgroups with distinct clinical outcomes and potential therapeutic implications.
The study showed that SB-509 was safe and well-tolerated, with no dose-limiting toxicities observed. Improvements in pain, numbness, and neurological symptoms were seen in approximately 50% of patients, with significant anecdotal improvements reported in neurologic exam scores and electrophysiologic testing.
Researchers at Cedars-Sinai Medical Center have developed a novel gene therapy strategy that modifies the brain microenvironment to induce an effective immune response against deadly brain tumors and infections. The treatment uses Flt3 Ligand to recruit antigen-presenting cells, which can destroy tumors and combat brain infections.
Researchers discovered that human cells deficient in LRP6 become resistant to anthrax toxin, while antibodies targeting LRP6 protect cells from toxicity. This finding suggests potential new avenues for treating late-stage anthrax disease.
Scientists at the University of California, Santa Barbara, have created a new lipid molecule that delivers therapeutic genes directly to cells, potentially helping inherited diseases and cancers. The novel molecule has a tree-shaped headgroup and displays superior DNA-delivery properties.
A research group identified 679 genes differently expressed in responders to chemotherapy compared to non-responders. RT-PCR analysis validated the findings, confirming the differential expression of 22 genes. The study suggests integrating molecular networks could improve diagnosis and treatment of colorectal cancer.
Researchers replaced a faulty gene responsible for hemophilia with a transposon from fish, preventing profuse bleeding in neonatal mice. The approach holds promise for treating the most common form of hemophilia, affecting nearly 18,000 Americans.
Defibrillator therapy found to be beneficial in reducing the risk of sudden cardiac death and prolonging life among patients with high-risk genetic cardiac disorders. The treatment is associated with significant cost savings, ranging from $15,000 to $20,000 per quality-adjusted-life-year saved.
Researchers at Florida State University have identified a gene called BDNF as playing a key role in social aversion, leading to the development of a potential new treatment for depression. Long-term use of antidepressants was successful in reversing social withdrawal, but a gene therapy approach showed promise with fewer side effects.
Researchers have identified a key gene associated with rare but important pregnancy disorders, including molar pregnancies, spontaneous abortions, and stillbirths. The discovery has significant implications for developing new treatments and therapies for these conditions.
A new lentiviral vector combines multiple gene manipulation techniques to efficiently regulate gene expression in cells. This versatile tool has potential applications in studying human genetic diseases, cancer research, and tissue engineering.
A study of 626 patients found that Black patients on HAART had a lower risk of atheroma than White and Hispanic patients. Genetic variants were associated with dyslipidemia in Hispanic patients, but not in others.
Researchers at Northwestern University have developed a gene therapy that turns off the alpha-synuclein protein, a key player in Parkinson's disease. The therapy uses RNA interference to selectively disable the gene, leaving other genes unaffected, and has shown promising results in rat models.
A saltwater aerosol solution has been found to restore the thin lubricant layer on airway surfaces, promoting mucus clearance and reducing lung damage in cystic fibrosis patients. The therapy is inexpensive and shows promise for slowing disease progression and improving patient outcomes.
A major gene therapy advance for treating Parkinson's disease has been funded by the Fox Foundation grant, with RheoGene Inc. developing a technology to manage gene expression. The therapy uses a patented small-molecule mediator to turn genes on or off, offering a potential solution to replacing lost dopamine in the brain.
The study illustrates 12 disease pairs and their therapeutic implications, including the use of severe caloric or protein restriction to treat diseases such as kidney failure and morbid obesity. Researchers propose considering genetic, infectious, and metabolic influences when looking for treatments, particularly in regard to HIV/AIDS.
Researchers used gene therapy to introduce a healthy copy of the dystrophin gene into mice with muscular dystrophy, repairing the entire muscle cell. The technique, developed by Michele Calos, has potential for long-term fixes for various genetic diseases.
Researchers at Memorial Sloan Kettering Cancer Center have successfully treated sickle cell disease in human models using stem cell-based gene therapy with RNA interference. The treatment corrected the production of abnormal hemoglobin and restored normal blood flow, offering new hope for patients.
Cedars-Sinai researchers have developed a new delivery system that can effectively regulate therapeutic gene expression, overcoming obstacles in bringing genetic therapies to humans. The system allows for the flexibility to turn gene expression on or off, even in the presence of an immune response, making it a critical tool for treatin...
Research confirms that individuals with a specific PTPN22 polymorphism are more likely to develop rheumatoid arthritis, with anti-CCP antibodies also playing a key role. The combination of these factors strongly predicts the onset of RA with a specificity of 100% for the disease.
Scientists have identified 570 genes associated with Parkinson's disease, which could lead to the development of new treatments. The study analyzed brains from 23 patients and found that limiting the activity of these genes may help control or even stop the progression of the disease.
Researchers at Children's Hospital of Philadelphia developed a novel technique to attach therapeutic genes to bare metal stents, reducing inflammation and restenosis. The study used animal models and found significantly lower restenosis with the new stents compared to conventional ones.
Researchers have found that Sangamo's ZFN-modified cells are resistant to HIV infection, whereas control cells are infected. The treatment works by disrupting the CCR5 gene, a receptor required for HIV entry into immune cells. This approach has advantages over other drugs in development, which require constant antagonist presence.
Researchers at Gladstone Institutes have made a breakthrough discovery about the role of miRNA-1 in the early stages of heart development. They found that miR-1 helps determine heart progenitor cells and maintain them until later embryonic stages, which could lead to new strategies for cardiac regenerative medicine.
Researchers have successfully treated hereditary spastic paraplegia by delivering a normal paraplegin protein to spinal motor neurons via gene therapy. This approach improved motor function in mice and holds promise for treating other forms of peripheral nerve damage caused by genetic mutations.
Research reveals that highly stimulated brain cells reactivate dormant survival genes, making them healthier and more resilient. This discovery holds implications for developing therapies to halt neurological diseases like Alzheimer's and Parkinson's, as well as mitigating the effects of maternal substance abuse on unborn babies.
Researchers at the University of Pennsylvania School of Medicine have discovered a human DNA-associated protein called LEDGF that controls where HIV integrates into human chromosomes. This finding has significant implications for improving the design of gene-therapy delivery systems.
A study of 5,700 patients found that carriers of a specific genetic variation had a 43% higher risk of death, heart attack, or stroke. The research aims to tailor drugs to individual patients based on their genetic makeup, potentially leading to safer and more effective treatments.
Researchers have engineered mice to respond to a therapy that lowers beta amyloid production, which forms senile plaques in the brain. Early treatment may be crucial in preventing plaque growth and improving outcomes for patients with Alzheimer's disease.
Researchers at Ohio State University discovered a new gene marker, ERG, that signals an aggressive form of acute myeloid leukemia (AML) requiring intensive therapy. High ERG activity is associated with poor relapse and survival rates in AML patients with normal cytogenetics.
Researchers found that exercise combined with gene therapy significantly extended the lifespan of mice with ALS, averaging 210 days, compared to 120 days in untreated mice. Early exercise initiation also improved outcomes, suggesting a potential strategy for slowing disease progression.
Dr. Robbins' research focuses on understanding the cellular functions of the heart and identifying genes associated with heart disease. He has developed genetically modified laboratory models to study the connection between protein mutations and heart disease.
Researchers have identified a specific FCGR3A-FCGR3B haplotype associated with an increased risk of developing rheumatoid arthritis. Homozygous individuals with this haplotype are three times more likely to develop RA, and those with the shared epitope protein sequence have a 10 times higher risk.
New treatments for Leber congenital amaurosis have been developed in mice, providing effective means of restoring retinal function. The treatments combine gene therapy and oral administration of vitamin A-like compounds, showing promise for treating this blindness-causing eye disease.
Researchers have successfully tested combining two treatments to restore retinal function in a mouse model of human inherited blindness. The combination of oral retinoids and gene therapy offers effective means for treating this devastating disease, with potential applications for wider age ranges of patients.
Researchers developed a gene therapy using adeno-associated virus (AAV-8) to deliver a normal copy of the delta-sarcoglycan gene to skeletal and cardiac muscle cells. The treatment showed remarkable improvements in muscle function, structure, and endurance in hamsters with muscular dystrophy.
The National Institutes of Health (NIH) has joined forces with advocacy groups to fund research on autism susceptibility genes. The initiative aims to expand knowledge of the genetic factors involved in autism and develop targeted therapies.
Researchers found that gene therapy using manganese superoxide dismutase plasmid liposome (MnSOD-PL) protected healthy tissue from damage during and after radiation retreatment. The therapy significantly improved survival rates and reduced side effects in lung cancer patients.
Researchers at Mount Sinai School of Medicine have developed a new gene therapy technique that permanently corrects genetic diseases like PKU by inserting genes into specific sequences between existing genes. The technique was tested on mice and successfully cured the disease with just three intravenous injections.
Researchers found that specific versions of the TGFb1 gene are linked to worse lung disease in cystic fibrosis patients. The study's large size and robust design enabled scientists to identify a potential target for improving CF therapy.
Researchers discovered that KLF2 regulates embryonic globin genes and maturation of red blood cells in a mouse model, potentially paving the way for future gene therapies. The study highlights the importance of understanding gene regulation in blood disorders like sickle cell anemia and beta-thalassemia.
Researchers identified genetic signatures that predicted patient prognosis and response to treatment, outperforming existing clinical markers. The technique of DNA microarray analysis holds promise for developing new prediction methods, sparing patients from unnecessary treatments.