Articles tagged with Gene Therapy
Researchers at Queen Mary University London have discovered a genetic defect causing kidney and adrenal disease in patients from around the world. The study found that defects in the SGPL1 gene can lead to progressive diseases with neurological involvement.
Dr. Stanley T. Crooke presents a detailed look at the mechanisms underlying antisense drug activity, including prehybridization, hybridization, and post-hybridization phases. He highlights his group's contributions to characterizing ASO therapeutics' design, targeting, and mechanism of action.
A recent study has identified 83 DNA variants that modulate human height, with some affecting it by more than 2 cm. The discovery is significant for understanding the genetic basis of complex diseases such as diabetes and schizophrenia, and may lead to the development of new therapeutic strategies.
Researchers developed a new gene-editing method called homology-independent targeted integration (HITI) that efficiently inserts DNA into genes in dividing and non-dividing cells of living rats. The technique uses the CRISPR-Cas9 tool to cut DNA at a specific location, followed by the NHEJ repair pathway for insertion.
Researchers developed a mini-intronic plasmid (MIP) system that can enhance transgene expression by up to 40-100-fold when used with adeno-associated viral (AAV) vectors. This could reduce the cost of gene therapy and lessen immune responses.
A new gene therapy has demonstrated effectiveness in clearing glycogen buildup from muscles in mice, potentially replacing enzyme infusions. The therapy uses a modified virus to deliver a gene that produces an enzyme missing in people with Pompe disease.
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 found that inhibiting PLSCR1 controls the infected cell's antiviral response, providing long-term protection from immune attack and excessive inflammation. This discovery holds promise for virally delivered treatments, inflammatory conditions, autoimmune disorders, and neurodegenerative diseases.
Scientists identified collateral lethal vulnerability in pancreatic cancers that can be targeted pharmacologically in certain patient populations. ME3 inhibitors may provide an effective therapy for many cancer patients.
Researchers found a significant excess of mutations in five genes previously implicated only in rare forms of epilepsy in individuals with common forms of the disorder. The study identified these genes as contributing to epilepsy risk in approximately 8 percent of people with familial non-acquired focal epilepsy.
Researchers at Northwestern University have discovered a genetic driver of mixed lineage leukemia, a rare and deadly form of childhood leukemia. They identified a targeted molecular therapy that halts the proliferation of leukemic cells by stabilizing the wild-type MLL protein, which drives cancer.
Scientists at Roswell Park Comprehensive Cancer Center have identified gatekeeper genes that allow prostate cancer to progress and resist treatment. The study highlights opportunities to prevent or reverse this process, offering new insights into lineage plasticity and its application in other types of cancers.
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.
This study investigates molecular changes in the OTOF gene in patients with auditory neuropathy and identifies the c.35delG mutation in the GJB2 gene. Cochlear implants show excellent outcomes, with improved speech development and language acquisition in patients with severe/profound hearing loss.
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.
Researchers have identified rare genetic variants linked to observable physiological characteristics in over 50,000 individuals. This approach has the potential to inform healthcare and prevent undiagnosed single-gene disorders like familial hypercholesterolemia.
Researchers identified five genes that, when inactivated, protected cells from HIV infection without affecting cellular survival. The genes target human proteins essential for HIV replication and viral entry into CD4 T cells.
Researchers used CRISPR-Cas9 gene-editing technology to identify three promising new targets for HIV infection. The study screened human genes essential for HIV replication but not for cellular survival, and identified five genes with potential therapeutic applications.
Researchers identified four new markers of primary sclerosing cholangitis risk on the human genome, bringing the total number of known predisposing locations to 20. The study also shed light on the genetic relationship between primary sclerosing cholangitis and inflammatory bowel disease.
Researchers at the University of Rochester Medical Center have identified a potential new treatment approach for lysosomal storage disorders, which cause disruptions in cellular functioning. The study found that repurposed drugs can overcome toxic build-up in affected cells, improving survival rates and quality of life.
Researchers used whole exome sequencing to analyze nearly 7,400 patients, identifying a genetic cause in 28 percent. The study shows that multiple genes can be involved in complex diseases, leading to imprecise diagnoses. A unified analysis combining clinical and genetic features provides more precise diagnoses.
Researchers investigated whether genetic variation in an immune system component called the complement system may contribute to vision loss during anti-VEGF therapy in wet AMD patients. Genetic variations were found to lead to complement system dysregulation, which was enhanced by blocking VEGF signaling.
Researchers reported the highest and most sustained levels of clotting factor IX in hemophilia B patients after a single dose of experimental gene therapy. Patients were able to discontinue factor infusions and engage in normal activities without disabling bleeding episodes, significantly improving their quality of life.
Researchers at Kazan University have developed a novel gene therapy approach that combines demineralized bone with genetic material to stimulate blood vessel and bone growth. The treatment shows promise in treating pseudarthrosis and other bone defects, with plans to offer innovative treatments at the Kazan University Clinic.
IDIBELL researchers describe the Ewing's sarcoma methylation profile for the first time, unveiling the potential of the PTRF gene as a prognostic marker and possible future therapeutic target. The study found that PTRF expression is associated with better survival rates in patients.
Researchers have discovered new compounds that are more stable and effective than their natural equivalents, leading to a better understanding of protein biosynthesis in cells. This could help design better therapeutics and improve patient outcomes.
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...
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.
The Center for Regenerative Medicine at Boston Medical Center and Boston University School of Medicine has received three prestigious NIH awards totaling over $9 million to further its commitment to induced pluripotent stem cell (iPSC) research and education. These awards will support the development of stem cell-based treatments for v...
Researchers at Columbia University Irving Medical Center have demonstrated that vision loss associated with retinitis pigmentosa can be slowed dramatically by reprogramming the metabolism of photoreceptors. The treatment aims to correct downstream metabolic aberrations, rather than the underlying genetic defect.
Researchers identified three gene expression signatures that can help rheumatologists predict which patients are more likely to respond to certain therapies. The findings could lead to higher response rates and reduced likelihood of ineffective treatment, ultimately improving patient care.
Researchers found distinct monocyte gene expression signatures in RA patients with different responses to anti-TNF therapy, potentially leading to personalized treatment approaches.
Researchers have discovered a new high-risk subtype of acute lymphoblastic leukemia (ALL) characterized by chromosomal rearrangements involving the MEF2D gene. A possible targeted therapy, panobinostat, has been shown to stop proliferation of human leukemic cells with this rearrangement.
Researchers develop domain-swapped T cell receptors that increase the safety of TCR gene therapy by preventing mispairing between introduced and resident chains. These new receptors retain functional domains and prevent autoimmune disease in mice and human cells, offering a promising tool for cancer treatment.
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.
Researchers at the University of Manchester have identified two gene mutations that trigger a rare retinal disease causing blindness in males. These findings offer promising insights into the development of gene therapy and potential treatments for X-linked Retinoschisis, a genetic disease leading to macular degeneration.
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 new human immunodeficiency has been discovered due to a faulty RASGRP1 gene, affecting T cells, B cells, and Natural Killer cells. The study identified a potential treatment using the drug lenalidomide, which reversed some effects of the mutation.
Researchers discovered a rare genetic variant in TM2D3 linked to late-onset Alzheimer's disease among Icelandic participants. The variant is estimated to increase the risk of Alzheimer's by approximately six times.
A Phase 1 clinical trial demonstrated the long-term safety of gene transfer in treating neovascular age-related macular degeneration. The therapy produced therapeutic proteins encoded by genes for at least 2.5 years in patients with advanced NVAMD.
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.
Blocking furin protein may offer unexpected benefits for patients with hemophilia A. Gene therapy and protein replacement treatments may be more effective with this approach.
Researchers at Binghamton University found that a genetic polymorphism in the CD40 gene modifies the severity of Duchenne muscular dystrophy, pointing to potential therapeutic approaches. The study suggests that drugs targeting CD40 may improve patient symptoms.
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 have engineered a novel, virus-like shell that can self-assemble and carry therapeutic DNA or RNA into human cells. This breakthrough could advance gene therapy treatments for rare genetic diseases and common conditions like diabetes.
Researchers at Rosalind Franklin University and Oregon Health & Science University developed a breakthrough study on treating congenital diseases in utero using antisense oligonucleotides injected into the amniotic cavity. The procedure resulted in targeted alteration of gene expression for up to a month after birth.
Boston University researchers have identified a new strategy to treat colon cancers driven by the mutant KRAS gene. The study found that inhibiting MEK and TAK1 genes suppresses the growth of these cancer cells, providing a potential avenue for precision medicine.
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 CHD1-depleted human prostate cancer cells are hypersensitive to DNA breaks and chemotherapeutic drugs, including PARP inhibitors. This suggests CHD1 as a potential biomarker for targeted prostate cancer therapy.
Researchers at Penn University have found remarkable similarities between human Leber congenital amaurosis and canine blinding disease Senior Løken Syndrome. The diseases share the same causative gene, NPHP5, and display similar pathology. The study's findings offer promising results for developing therapies to treat these conditions.
The Generation Study will enroll over 1,300 cognitively healthy older adults at high risk of developing Alzheimer's due to inherited APOE gene mutations. Researchers will test two investigational treatments: an active immunotherapy and an oral medication to stop amyloid accumulation.
Researchers have developed a gene therapy approach using ultrasound energy and microbubbles to selectively open cells, allowing delivery of therapeutic agents. The study's findings will help refine this technique as a clinical tool and translate it into an effective gene or drug delivery tool for patients.
Researchers at UC Berkeley discovered a way to boost CRISPR-Cas9 cutting efficiency up to fivefold by disrupting DNA repair mechanisms with short oligonucleotide pieces. This technique increases the success rate of creating knockouts, essential for studying gene function and correcting hereditary mutations.
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.