Articles tagged with Gene Therapy
The study highlights the importance of protease-activated receptors (PARs) in cancer growth and development, with PH-binding motifs identified as a key platform for drug design. The researchers suggest that targeting PARs could provide an alternative to current oncogenic pathways.
Researchers discuss the benefits of CAR-T therapy in treating B-cell lineage acute lymphoblastic leukemia (B-ALL) in children. The therapy, tisagenlecleucel, has shown promising results and is now priced at $508,250, a more manageable cost compared to other gene therapies.
Researchers have engineered an AAV that efficiently crosses the blood-brain barrier and delivers genes to the brain in humanized mice. This could lead to new treatments for severe genetic brain disorders with no current cures or few treatment options.
A Japanese research team used machine-learning-driven modular assembly systems to create a more efficient gene editing tool. The study demonstrated an improvement in genome editing efficiency by 5%, showcasing the effectiveness of engineering zinc-finger nucleases through structural modeling.
A new study shows that gene therapy delivered by nanocarriers can repair damaged discs and reduce signs of back pain in mice. The treatment, which uses naturally derived nanocarriers to deliver genetic material for a protein key to tissue development, restored structural integrity and function to degenerated discs.
A study utilizing RNA-Seq and single-cell RNA-Seq data identified key transposable elements (TEs) linked to myocarditis. These findings reveal a significant role for TEs in modulating immune responses, providing new insights into cardiomyopathy's pathogenesis.
The Ottawa Hospital is receiving a $59 million grant to boost Canada's capacity for life-saving biotherapeutics, including vaccines, gene therapies, and cell therapies. The funding will support the construction and operation of a world-class biomanufacturing facility at its new campus.
Researchers at the University of Trento have identified a collection of molecular tools to rewrite DNA, including a compact Cas9 enzyme from the human microbiome. The discovery has potential for gene therapy applications and could speed up the development of therapies for genetic diseases.
Researchers at UW–Madison developed a method using short electric pulses to increase gene therapy material uptake in liver cells, reducing dosage needed. The technique could help treat genetic diseases like cystic fibrosis and sickle-cell disease.
Researchers identified a spectrum of effects on sodium channel function due to SCN2A variants, with hyperactive channels linked to early seizure onset and underactive channels associated with autism. The study provides insights into the relationship between genetic changes, disease severity, and age of seizure onset.
A phase 3 clinical trial found that 96.7% of patients did not experience severe vaso-occlusive crises, and all remained hospitalization-free for at least one year after receiving the gene therapy CASGEVY.
Researchers develop gene therapy to delay progression of metachromatic leukodystrophy by correcting enzyme deficiency and reducing neuroinflammation. Successful treatment has been demonstrated in mice, paving the way for potential human clinical trials.
Researchers have identified a novel protein FOXF1 that stabilizes blood vessels inside lung tumors, decreasing intertumoral hypoxia and preventing lung cancer metastases. Increasing levels of FOXF1 or FZD4 shows promise to improve therapeutic outcomes in lung cancer patients.
A study in the Journal of Neuromuscular Diseases found that disease-modifying gene therapy treatments improve motor function, bulbar function, and pulmonary function in infants with spinal muscular atrophy. The real-world data from a large patient registry confirms improved safety profiles for early treatment opportunities.
Researchers have discovered a new immunotherapy approach to overcome resistant leukemia by targeting the mutated TP53 gene. Combining pharmacological therapies with genetically engineered CAR T-cells increases effectiveness against cancer cells, offering promising strategies for patients with resistant disease.
A clinical trial at NIH demonstrated that experimental gene therapy slowed loss of motor function in patients with giant axonal neuropathy, a fatal childhood disease. The treatment also showed signs of regeneration in sensory nerves, offering new hope for those affected.
Scientists have successfully tested novel gene therapies in a whole human liver, opening up new avenues for treating life-threatening inherited diseases. The use of a normothermic liver perfusion system enables accurate testing of gene therapeutics directly in the clinical target organ.
Christiana Wang, a second-year PhD candidate, has been awarded the prestigious award for her platform presentation on antisense oligonucleotide therapy for a dominant negative SPTAN1 pathogenic variant. Her research aims to develop individualized therapy for treating rare genetic disorders.
Meena Sethuraman, a third-year medical student, received the 2024 ACMG Foundation/Revvity Travel Award for her research on genetic variants in fatty acid oxidation disorders. The award recognizes her platform presentation on characterizing pathogenicity of ACADVL variants in very long-chain acyl-CoA dehydrogenase deficiency.
Scientists from the University of Cologne developed threofuranosyl nucleic acid (TNA) with a new base pair, offering improved stability and function compared to natural DNA and RNA. This breakthrough could enable targeted drug delivery, diagnostics, and recognition of viral proteins or biomarkers.
Researchers at the University of Cincinnati Cancer Center have identified a new protein called p47 that helps prevent breast cancer metastasis. The study found that lower p47 expression was correlated with higher breast cancer metastasis, and that increasing p47 function could potentially lead to new therapies.
Researchers have discovered two genetic variants associated with generalized pustular psoriasis (GPP), a rare and serious condition characterized by widespread skin lesions and inflammation. These variants, found in the MEFV gene, may hold promise for new diagnostic and therapeutic approaches to GPP.
UCSF scientists discover delivering therapeutic molecules to amniotic fluid can effectively treat Angelman syndrome and other neurological conditions. The treatment uses antisense oligonucleotides, which can alter gene expression, and has shown improved motor function and learning outcomes in mice.
Recent clinical trials highlight a new challenge in AAV gene transfer: complement system activation, which may contribute to adverse events and immune priming. The article provides a comprehensive review of the complement cascade and its interactions with AAV vectors.
A new approach to fighting HIV has been developed using RNA, specifically small interfering RNAs (siRNA), which regulate gene expression in cells. This nanomedicine was shown to reduce HIV replication by 73% and is intended for vaginal application to prevent sexual transmission.
A new technique employing a retrotransposon from birds may provide a safer alternative to CRISPR-Cas9 gene editing by inserting genes into a designated 'safe harbor' in the genome. This approach could complement CRISPR technology and enable efficient gene supplementation for hereditary diseases.
Scientists have developed a single-dose genetic medicine that halts the progression of both ALS and frontotemporal dementia in mice. The treatment targets pathological TDP-43 build-ups in brain cells, which may also treat common forms of dementia like Alzheimer's disease.
A recent study has uncovered 145 genes crucial for genome stability, shedding light on genetic factors influencing human health over a lifespan. The research highlights the potential of SIRT inhibitors as a therapeutic pathway for cohesinopathies and other genomic disorders.
A study published in Oncotarget has identified specific mutational and therapeutic landscapes of pancreatic cancer in the Russian population. By applying machine learning models to full exome individual data, researchers received personalized recommendations for targeted treatment options for each clinical case.
Researchers found elevated PROX1 levels in advanced colon adenocarcinoma, correlating with poor prognosis. PROX1 modulates CRC cell behavior, influencing invasiveness and survival outcomes. The combined PROX1/α-SMA gene set emerges as a potential CRC prognostic marker.
Researchers identified senescence-related tumor microenvironment genes associated with poor prognosis, genetic alterations, and reduced responsiveness to immunotherapy in HNSC. The study highlights the importance of precision medicine approaches for personalized treatment.
A novel cyclic thiosulfinate cross-linker has favorable drug-like properties and can stabilize the SOD1 dimer in vivo, indicating potential therapeutic benefits for fALS. This study uses a mouse model to evaluate protein cross-linking as a strategy to treat SOD1 variants associated with familial ALS.
Researchers have found that a gene-based therapy targeting plakophilin-2 can interrupt the progression of arrhythmogenic right ventricular cardiomyopathy, a rare inherited disorder. The treatment reduced episodes of arrhythmia and slowed the deterioration of the heart's walls in mice.
Researchers used fMRI to assess brain responses to lights stimulating only cone cells in dogs with different types of retinal diseases. The study found that gene augmentation therapy restored response in cortex to black and white stimulation, making this disease a promising one for photoreceptor cell replacement treatment.
A novel gene therapy has been shown to restore hearing function in six children with DFNB9, a form of autosomal recessive deafness. The treatment, which uses an adeno-associated virus carrying a version of the human OTOF gene, demonstrated significant improvements in speech perception and ability to conduct normal conversation.
A cross-sectional study of over 6,500 adults aged 65+ found that older adults spent an average of 20.7 days per year receiving healthcare outside the home. The 'health care contact days' metric can help evaluate care and identify areas for improvement, such as coordinating tests with visits and reducing Friday office visits.
Researchers identified genetic variants that predict response to treatment for preterm birth, a condition affecting one in 10 infants. High levels of mutations in certain genes are associated with lower response rates, suggesting a precision framework for future drug development.
A team of researchers identified a CTP-dependent transcription factor controlling Shigella virulence gene expression, providing new avenues for combating this and related bacterial pathogens. The discovery sheds light on the molecular mechanisms underlying bacterial pathogenesis.
Researchers identified key characteristics associated with improved CAR T outcomes in large B cell lymphoma, including a B-cell gene expression signature and high CD19 protein expression. Patients who received axicabtagene ciloleucel had better event-free survival compared to those receiving standard therapy.
Researchers studied the effects of resveratrol on circadian clock gene expression in young and older human adipose-derived progenitor cells. They found increased levels of some components in older-APCs compared to young-APCs, but also observed gained rhythmicity of some components after resveratrol treatment.
Rice bioengineer Jerzy Szablowski and colleagues have engineered a synthetic serum marker that enables non-invasive neural monitoring by tracking gene expression dynamics in the brain. This breakthrough allows researchers to investigate brain development, cognitive function and neurological diseases more effectively.
Researchers have successfully used mRNA technology to correct a rare genetic disease in mice, demonstrating its potential therapeutic use. The treatment corrected the lethal consequences of the disease and restored glutathione metabolism.
Researchers at Baylor College of Medicine have developed a technology to effectively regulate gene expression, promising a safer approach to gene therapy. The system uses small molecules to interact with RNA, allowing for precise control over protein production and therapeutic window maintenance.
Researchers at UCL Queen Square Institute of Neurology have developed a new gene therapy that significantly reduces seizures in mice with focal cortical dysplasia. The treatment, which involves the overexpression of a potassium channel, has shown an average reduction of 87% in seizures without affecting the mouse's memory or behavior.
Researchers developed a gene therapy approach to target the root cause of ACM, restoring plakophilin-2 levels and improving heart function. Clinical trials starting in 2024 aim to explore this approach in patients with PKP2 mutations.
Gene therapy treatments can prompt an adverse immune reaction when the body mistakenly perceives the treatment as a viral threat. Researchers have identified a critical pathway in the liver that triggers this response, but also found a way to block it, paving the way for safer and more precise gene therapy options.
A team of researchers has developed a promising gene-editing strategy for spinal muscular atrophy (SMA), a devastating pediatric neuromuscular disorder. The approach involves using CRISPR base editing to activate the SMN2 gene, which is similar to the mutated SMN1 gene responsible for SMA.
Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting galectin 1 protein using gene therapy can shrink liver cancer in mice, improving the anti-cancer immune response and increasing killer T cells. This approach has potential as a treatment for HCC prevention.
Researchers at NTNU are developing a new therapy for rare hereditary diseases like DOOR syndrome using mini-brains grown in the lab. The treatment involves injecting a virus with a healthy OXR1 gene to produce the missing protein, which may help stop or reverse the disease.
Researchers discovered a link between gene therapy and the accumulation of stem cells with genetic mutations, which can lead to accelerated growth and increased blood cancer risk. The study suggests that younger patients may be safer candidates for gene therapy due to fewer genetic mutations.
A new review article highlights significant milestones in gene therapy for rare diseases, improving efficacy and safety. However, challenges remain to make this transformative approach widely available.
The Genes & Health study has enrolled its 10th participant in a gene-editing clinical trial for heart disease, specifically familial hypercholesterolemia. This milestone marks an important step towards improving health outcomes for people of Pakistani and Bangladeshi descent.
Researchers at Duke University developed a CRISPR-based platform to identify genes that improve T-cell therapies for cancer treatment. They discovered BATF3, a single master regulator of the genome, which reprograms thousands of genes in T cells and greatly enhances cancer cell killing.
Researchers created an immunodeficient mouse model of physiological ovarian aging, mirroring female fertility decline with age. The study found decreased oocyte quality, impaired follicle activation and growth, and increased mitochondrial dysfunctions in Old mice.
A novel gene therapy strategy has been developed to specifically target and activate the direct pathway in Parkinson's disease-affected neurons, leading to improved motor symptoms such as bradykinesia and tremor. The approach has shown faster onset and longer duration compared to traditional L-Dopa treatment.
Individuals with severe sickle cell disease express willingness to accept gene therapy risks for a potential cure. Gene therapies offer higher chances of eliminating symptoms and extending life expectancy, but also come with significant risks.
Researchers have developed a novel approach, REVeRT, to efficiently transport large genes using dual AAV vectors at the transcript level. This new method offers increased efficiency, fewer side effects, and greater flexibility compared to existing strategies.
Researchers developed the DANGER analysis tool to overcome challenges in CRISPR gene editing. The tool assesses on- and off-target effects without a reference genome, quantifying phenotypic risk at the gene ontology level. This breakthrough enables safer strategies for genome editing in various organisms.
Researchers at the University of Texas Health Science Center discovered a gene therapy approach that can restore motor functions in mice with Contactin-Associated Protein 1 (Cntnap1) mutations. The study found that turning on the normal gene earlier improves the rescue outcome, and the next phase is to test this approach in humans.
In a phase I trial, an oncolytic virus treatment designed by Brigham researchers extended survival among patients with recurrent glioblastoma, especially those with pre-existing viral antibodies. The therapy reshaped the tumor's surrounding environment to stimulate an anti-tumor immune response.