Articles tagged with Gene Therapy
Researchers have developed a gene therapy that improves muscle strength, corrects muscle structure, and prolongs life in animal models of X-linked myotubular myopathy. This devastating disease affects approximately 1 in 50,000 male births, causing severe respiratory difficulties and requiring intensive support.
Researchers from Penn Vet found a shared cell death pathway involved in inherited vision diseases, including rod cone dysplasia and X-linked progressive retinal atrophy. The study suggests that targeting this pathway with TNF-inhibitors could slow disease progression or complement gene therapies.
Researchers have developed a gene therapy treatment that improves vision in people with choroideremia, a condition causing progressive loss of sight. The treatment showed significant improvements in visual acuity and sensitivity to light in six patients, offering promise for future treatments of other retinal diseases.
Researchers found that mda-9/syntenin is a driving force behind glioblastoma's growth and invasion. The study identified molecular targets that could lead to new therapies and potentially use the gene to monitor disease progression.
A recent study published in Human Gene Therapy evaluated the immune response to viral vector delivery systems and their impact on gene therapy's safety and efficacy. The researchers found that the systemic and local immune reactions induced by AAV-based gene therapy did not affect the safety of gene therapy or expression of the replace...
Researchers found that a small number of transplanted stem cells was sufficient to restore normal skin function without adverse side effects. The treatment resulted in long-term restoration of normal skin, even after 80 cycles of renewal.
A comprehensive genomic analysis of cervical cancer identified recurrent genetic mutations, including one targeted by existing treatments for other cancers. The study also shed light on the role of human papillomavirus (HPV) in cervical cancer development.
An international group of investigators has discovered new genetic variations and cell types involved in inherited susceptibility to rheumatoid arthritis. The study provides a definitive list of major common genetic variation involved in the disease, leading to potential new therapies.
Researchers have developed a technique combining cell transplantation and gene therapy to target BACE1, the key enzyme in amyloid beta protein production. This approach reduces Aβ protein production by downregulating the BACE1 gene, offering new therapeutic avenues for Alzheimer's disease.
A new study describes a mechanism by which microRNAs are encapsulated and exported in nanovesicles, facilitating intercellular communication. The researchers identified EXOmotifs, specific nucleotide sequence patterns, that enable the export of miRNAs from human T lymphocytes.
Researchers have developed a nonsense suppression drug that can restore function in mice with aniridia, a progressive disease affecting the eyes and brain. The treatment, ataluren, was administered topically to postnatal aniridia mice and successfully reversed eye deformations and restored vision.
Researchers discovered that six autoimmune diseases arise from DNA changes located outside genes. Multiple genetic switches controlling gene expression are affected in each disease, leading to incorrect gene expression. The study provides a new model for understanding the genetic causes of common complex diseases.
Researchers discovered a genetic variant that influences vaccine response and autoimmune disease risk. Individuals with this variant express an additional immune system receptor on their B cells, which activates antibody production, potentially leading to autoimmune diseases.
Researchers discovered a genetic variation that predisposes mice to severe, inflammatory arthritis. The study implicates a lysosomal enzyme as a contributor to the development of the disease.
Dr. Katherine High's groundbreaking research in gene therapy has led to novel approaches to correcting hemophilia, with ongoing studies in a current NIH-funded clinical trial. The E. Donnall Thomas Prize recognizes her remarkable contributions to the field, transforming the notion of using genetically engineered mechanisms for treatment.
Research found that women with HER2- and hormone receptor-positive breast cancer treated with neoadjuvant therapy had lower pathologic complete response rates if their tumors had PIK3CA gene mutations. These mutations were more common in HER2-positive breast cancers.
A new gene therapy has led to a dramatic decline in bleeding events in dogs with naturally occurring hemophilia A. The treatment involves packaging specialized blood platelets with genes that express clotting factor, resulting in significantly fewer serious bleeding events over three years.
A new study demonstrates the effectiveness of antisense drugs in targeting a wide range of tissues and organs, including liver, kidney, lung, muscle, and peripheral nerves. The findings suggest that antisense therapeutics have broad therapeutic potential for various disease indications.
Researchers at A*STAR's Institute of Medical Biology and Institute of Molecular and Cellular Biology have identified a gene encoding a hormone called ELABELA, which is crucial for heart formation in zebrafish embryos. The discovery holds great promise for the development of targeted therapies for heart disease and blood pressure control.
A study found that cognitive behavioral therapy for PTSD reduces symptoms and affects underlying biology, reversing changes in gene expression and brain volume. The therapy was associated with improved symptoms and increased hippocampal volume and FKBP5 gene expression.
St. Jude Children's Research Hospital scientists have identified an enzyme that can halt or reverse the build-up of toxic protein fragments in mice with Alzheimer's disease. Gene therapy increased activity of the enzyme neuraminidase 1, reducing plaques in the brain involved in learning and memory.
A new study published in the New England Journal of Medicine found that a gene-based method for selecting warfarin doses has no added benefit over standard dosing methods. However, African Americans patients showed better outcomes with non-genetic strategy, highlighting the need for further research on personalized medicine approaches.
A single dose of AAV1/SERCA2a gene therapy has long-lasting benefits for advanced heart failure patients, improving heart function status and reducing recurrent cardiovascular events. The study results support the potential use of this gene therapy as a new tool for treating advanced heart failure.
Researchers found that a gene therapy approach using rAAV vectors can promote a regulatory immune response, allowing for sustained gene expression in muscle tissue without the need for immunosuppression. The study used M-type α-1 antitrypsin (M-AAT) and demonstrated long-term expression in patients with AAT deficiency.
Researchers at KU Leuven developed a new protein fusion that combines HIV and MLV replication proteins to create a safer viral vector for gene therapy. This fusion protein reduces the risk of cancer-causing mutations when integrated into human cells, paving the way for more effective treatments for blood diseases.
A new gene therapy has shown promising results in reversing heart failure by delivering the SUMO-1 gene directly to the heart. In preclinical testing, the therapy improved cardiac function, reduced heart volumes, and enhanced blood flow compared to other treatments.
Researchers from Arizona State University and Stanford Universities have discovered that the key gene Numb plays a role in promoting muscle growth and suppressing Myostatin, which limits muscle growth. The findings provide a new avenue for developing treatments for muscle diseases such as muscular dystrophy and ALS.
The GIANT trial found that identifying slow responders to clopidogrel through genetic profiling and adjusting treatment leads to reduced adverse events and similar ischemic event rates compared to those with a favorable genetic profile. This suggests alternative treatment strategies for patients identified as CYP2C19 poor metabolizers.
A Northwestern University research team has developed a drug that silences a critical gene in glioblastoma, increasing survival rates by nearly 20% and reducing tumor size. The novel therapeutic uses nanotechnology to target the gene, which plays a key role in therapy resistance.
Researchers at Nationwide Children's Hospital have found a way to overcome the immune system's attack on gene therapy viruses. By using plasmapheresis, a process that temporarily removes antibodies from the body, they were able to increase gene expression in animals with Duchenne muscular dystrophy by 500 percent.
A global collaboration of researchers has identified 11 new genetic areas of interest contributing to late onset Alzheimer's Disease. The study expands the scope of disease understanding to include new areas such as the immune system, where a genetic overlap with other neurodegenerative diseases was found.
Researchers discovered 11 new regions of the genome that contribute to late-onset Alzheimer's disease, doubling the number of potential genetics-based therapeutic targets. The study also identified a genetic overlap with other neurodegenerative diseases, including multiple sclerosis and Parkinson's disease.
Researchers at the University of Cambridge have discovered that genetic mutations in the KSR2 gene can lead to severe obesity and reduced basal metabolic rate. This finding provides evidence for the role of genes in regulating energy use in the body, adding complexity to the body's mechanisms for regulating weight.
Acute dendritic cell leukaemia has a poor prognosis due to its rarity and difficulty in treatment. Researchers sequenced the exome of three patients and found mutations in epigenetic genes, which could lead to new therapeutic approaches.
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.
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 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.
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.