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 meta-analysis of randomized controlled trials found that chemotherapy improved progression-free survival compared to epidermal growth factor receptor tyrosine kinase inhibitors in patients with wild-type EGFR tumors. However, overall survival did not differ between the two groups.
A gene therapy approach using adeno-associated virus (AAV) successfully treated heart disease in mice with Friedreich's ataxia, restoring full functionality to the hearts. The treatment, which introduced a normal copy of the FXN gene, prevented the development of heart disease and even fully cured advanced-stage cases.
Researchers aim to deliver GDNF into muscle cells using a viral vector to protect motor neurons and slow disease progression in a rat model of ALS. The study, led by Cedars-Sinai Regenerative Medicine Institute, could pave the way for a clinical trial if successful.
A new gene therapy has shown promising results in improving hind limb function in rats with spinal cord injuries. The treatment involves delivering a scar-busting gene that promotes the survival of nerve cells and reduces inflammation, leading to improved mobility and tissue repair.
Researchers at Tel Aviv University have discovered that DNA therapy can preserve inner ear nerve cells in humans with certain types of progressive hearing loss. The study uses a protein growth factor to block degeneration and has important implications for enhancing sound perception with cochlear implants.
Marina Cavazzana and Adrian Thrasher have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders. They are pioneers in treating life-threatening inherited diseases of the immune system with gene therapy, using a patient's own modified stem cells.
Researchers used a rat genomic gene-chip to profile hippocampal gene expression changes after electroacupuncture therapy. The results showed that electroacupuncture regulates the expression of specific genes involved in depression, including Vgf and Igf2. These findings suggest that electroacupuncture may be a useful treatment for depr...
Researchers found that patients with specific gene variants exhibited a significantly lower risk of experiencing relapse after treatment for chronic hepatitis C. The study, which involved over 300 patients, suggests that these variants may play a role in preventing the virus from becoming unstable and causing recurrence.
Researchers at Sanford-Burnham and Icahn School of Medicine at Mount Sinai have discovered a key cellular process leading to heart failure, which can be halted with a therapeutic approach. Blocking the effects of miR-25 has improved cardiac function and survival in mice.
A new gene therapy approach has been shown to be safe and well-tolerated in a clinical trial of four children with mucopolysaccharidosis type IIIA, an inherited lysosomal storage disease. The treatment involved delivering therapeutic genes via an adeno-associated viral vector, resulting in improved brain shrinkage and behavioral changes.
Genethon, a non-profit organization, has partnered with Esteve to manufacture the gene therapy AAV9-hsulfamidase for treating Sanfilippo syndrome. This agreement supports clinical development and advances the treatment towards trials.
A research team developed an adjuvant that optimizes lentivirus gene transfer by enhancing virus attachment to target cells, resulting in a three-fold increase in transduction rate. This improvement reduces the need for additional viruses, potentially leading to more effective treatments for genetic disorders.
Johns Hopkins researchers have identified a set of genes that can be turned back on to evade immune detection in ovarian, breast, and colorectal cancer. Reversing these epigenetic changes with an FDA-approved drug allows cancer cells to become more vulnerable to immune therapy, potentially leading to more effective treatment.
A study by Michigan State University researchers used rats and vanilla frosting to identify genetic factors contributing to binge eating. The Sprague-Dawley rat strain was found to be prone to binge eating, narrowing the scope of possible genes involved in the disorder.
The European Commission has invested nearly $475 million in 100 gene transfer projects to support basic and clinical research in the field of gene and cell therapy. This funding is aimed at developing new treatments for chronic and rare diseases, as well as novel regenerative medicine approaches.
Joseph C. Glorioso, III, PhD received a Pioneer Award from Human Gene Therapy for his leadership and accomplishments in developing herpes viruses as efficient vectors for delivering therapeutic genes into cells. His research has shown promise for treating complex genetic and acquired diseases, including brain degeneration and cancer.
Researchers have discovered that genes expressed in the nasal passages can serve as accurate proxies for those expressed deeper in the lungs. This breakthrough allows for less invasive and more affordable genetic profiling of asthmatic patients, potentially leading to improved treatment options and better understanding of the disease.
Duke researchers have successfully used gene therapy to induce stem cells to produce growth factor proteins, overcoming the challenge of delivering these proteins after implantation. The technique allows for long-term delivery and could be applied to various orthopedic tissues, presenting a significant step toward commercialization.
A team of researchers at the University of Montreal has discovered a promising new approach to treating leukemia by targeting the Brg1 gene. The study found that removing or inhibiting this gene can permanently shut down cancerous cell growth and division.
Hal Dietz, a Johns Hopkins pediatric cardiologist and geneticist, has been recognized for his groundbreaking work on Marfan syndrome. He identified the cause of the rare genetic disease and found that losartan can attenuate overgrowth of the aorta.
Researchers use antisense oligonucleotides to skip over the mutated exon causing Huntington's disease, preventing formation of toxic protein fragments. The innovative therapeutic strategy is a proof-of-concept for treating complex diseases.
Scientists have identified a genetic function called Grasp that plays a crucial role in maintaining the proper function of the p53 tumor suppressor gene. This discovery could lead to a new approach in cancer therapy by activating or stimulating the Grasp gene to enhance p53 function.
Dr. Ronald G. Crystal developed the first in vivo adenoviral gene delivery vector, accelerating gene therapy translation from lab to clinic. The Pioneer Award recognizes his seminal work on adenoviral vectors.
A collaborative effort between the GENEFA Platform and FARA has secured $100,000 in annual funding for a two-year research project to develop a gene therapy-based treatment for Friedreich's ataxia. The project aims to rescue a mutated frataxin gene defect in cells of the central nervous system.
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.
A new study found that combining high-dose interleukin-2 therapy with personalized tumor cell-based immunotherapy significantly improves patient survival rates. The treatment approach, which activates patients' immune systems using their own tumor cells, outperforms IL-2 alone in extending survival.
A study by Columbia University Medical Center researchers identified matrix metalloproteinase-9 (MMP-9) as a key factor contributing to motor neuron degeneration in amyotrophic lateral sclerosis (ALS). The findings suggest that MMP-9 inhibitors may offer a new therapeutic option for treating this incurable neurodegenerative disease.
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.
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.
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.
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.
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.
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 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.
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 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.
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.