Articles tagged with Gene Therapy
Researchers have developed a gene therapy that rescues cilia defects in retinal cells affected by Leber congenital amaurosis, a disease causing blindness in early childhood. The therapy restores opsin protein concentration in photoreceptor outer segments, suggesting functional NPHP5 may stabilize the primary cilium gate.
Breast cancer recurrence and metastatic spread remain a significant challenge, with researchers identifying a metabolic signature that can predict patient outcomes. The signature could be used to develop new therapies targeting cancer metabolism.
A novel stem cell-gene therapy has been shown to be safe in humans, with no serious side effects reported in the first trial. The treatment targets motor neurons that die in patients with amyotrophic lateral sclerosis (ALS), a fatal neurological disorder.
Researchers have developed a novel DNA-based method to identify people at greater genetic risk of developing Alzheimer's disease before symptoms appear. The method uses data on common DNA variants from over 7.1 million individuals and predicts a person's risk of Alzheimer's, depending on which DNA variants they have.
A new study identifies how the suppression of a specific transcription gene triggers changes that impair oligodendrocyte function in Huntington's disease. The researchers believe replacing or fixing defective glia cells may prove a far easier proposition than replenishing neurons lost in the disease.
A new preclinical model of thymic cancer reveals how a common mutation sparks tumor formation and identifies potential targets for therapy. The model, developed by Weill Cornell Medicine researchers, shares molecular characteristics with human thymic tumors, paving the way for new treatments.
Researchers from Trinity College Dublin have developed a new gene therapy approach targeting mitochondrial function to treat dry AMD, a debilitating eye disease. The therapy has shown benefit in multiple models of the condition, offering hope for a potential treatment that could help millions globally.
Researchers at UVA have discovered the mechanism behind gene regulation during organ development, shedding light on how genetic material interacts with transcription factors to create different cell types. The study's findings could offer insights into the initiation of certain cancers and inspire new therapeutic development.
A team of researchers has identified two compounds that target DNMT3A complexes in cancer cells, offering a safer and more targeted treatment for leukemia patients. The findings provide hope for reducing off-target toxicity associated with current leukemia therapies.
A new study has partly restored the function of retina's cone receptors in two completely colorblind children using gene therapy. The treatment has been shown to activate previously dormant communication pathways between the retina and brain, drawing on the plastic nature of the developing adolescent brain.
Researchers found that using the drug erlotinib, which blocks a specific cancer pathway, led to a 30 percent reduction in polyps formed in patients with FAP. The study's findings suggest potential ways to prevent colorectal cancer in the general population at an earlier stage.
Researchers found that administering tisagenlecleucel at the higher end of the approved dose range significantly improves overall and event-free survival in young patients. The study suggests that using higher doses of CAR-T therapy can achieve more effective and long-term responses without increasing toxicity risk.
A study published in Molecular Therapy — Methods & Clinical Development shows that delivering the protein EPS8 via gene therapy can rescue malfunctioning inner ear hair cells that transduce sound. In mice affected by recessive deafness, EPS8 increases stereocilia length and restores hair cell function.
A team of researchers from The Mount Sinai Hospital has made a groundbreaking discovery into the genetic and molecular mechanisms that predispose individuals to Alzheimer's disease. They identified 21 candidate risk genes, including SPI1, which regulates microglia and AD risk.
A new gene therapy approach using the neuroprotective protein SynCav1 has shown promising results in slowing down ALS disease progression and increasing life span in rodent models. The treatment preserved spinal cord motor neurons and extended longevity in mice, with similar effects observed in a rat model of ALS.
Researchers discover gene AVIL responsible for deadly brain tumor also causes two forms of childhood cancer, rhabdomyosarcoma. Blocking AVIL activity prevents formation of the disease in lab samples and mouse models.
Researchers used Guardant NGS to analyze nearly 17,000 lung cancer samples and found MET amplification in 1.2% of cases, with 20.8% having overlapping oncogenic drivers. The study suggests that high gene copy numbers and smaller amplified regions can be used to enrich for the true MET-sensitive population.
A study of over 500 patients with multiple myeloma reveals a high prevalence of genetic alterations in oncogenic pathways, leading to treatment resistance. The research found a specific link between RASopathies and mutations in these pathways, offering new insights into the development of resistance mechanisms.
A team of researchers from UMass Amherst and UMass Chan Medical School has developed a technique to increase the secretion of alpha-1 antitrypsin (AAT) in muscle cells by about 50 percent. This breakthrough will help improve gene therapies for diseases caused by dysfunctional protein production.
A new study from Tel Aviv University found that CRISPR therapeutics can lead to a significant loss of genetic material in treated cells, potentially destabilizing the genome and promoting cancer. The researchers detected up to 10% of cells with lost chromosomes, highlighting the need for extra care when using this technology.
Researchers have created a new DNA atlas that provides insights into how genes in specific cells contribute to coronary artery disease. The atlas identifies over 200 independent genetic markers associated with disease risk, offering a potential roadmap for interpreting non-coding variants.
A new study may help develop therapies to slow vision loss in pigmentary retinopathy by understanding how the visual system adapts to photoreceptor death. The research found that the visual pathway becomes hyperactive during early RP, which could lead to therapeutic protection and restoration of vision.
A new type of adeno-associated virus (AAV) gene therapy candidate, FLT180a, has been shown to reduce bleeding risk in patients with haemophilia B. The treatment led to sustained production of FIX protein from the liver in nine out of ten patients, eliminating the need for regular replacement therapy.
Researchers used deactivated Cas9 proteins to target key segments of the human genome and synthetically trigger gene transcription. The study revealed that enhancers can send messages in both directions, but with a predominant regulatory mode where an enhancer tracks toward corresponding promoters.
A world-first study has confirmed the link between Alzheimer's Disease and multiple gut disorders, revealing shared genetic architecture. The study suggests that abnormal cholesterol levels play a key role in both conditions, and may lead to new potential treatments for Alzheimer's and gut disorders.
Researchers found evidence of how destructive proteins attack human brain cells and destroy surrounding tissue. The study identified a pivotal mechanism that could be a potential therapy for the disease.
Researchers from Rice University, Duke University, Brown University and Baylor College of Medicine developed a magnetic technology to wirelessly control neural circuits in fruit flies. They used genetic engineering to express heat-sensitive ion channels in neurons that control the behavior, and iron nanoparticles to activate the channels.
A new treatment approach using antisense oligonucleotides (ASOs) may help reduce cystic fibrosis symptoms and improve quality of life for patients with a specific gene mutation. The ASO strategy tricks cells into making an imperfect but functional version of the CFTR protein, which is better than having none at all.
Researchers at Edith Cowan University have found a genetic link between human leukocyte antigens and immunotherapy side effects in non-small cell lung cancer patients. The discovery enables doctors to tailor treatment to individual patients, reducing the risk of toxicities and improving overall outcomes.
A study found that individuals with a rare genetic variant common in people of East Asian descent are at higher risk of vessel reclog during or shortly after endovascular therapy for ischemic stroke. The researchers analyzed data from 277 East Asian adults who received the treatment between 2011 and 2021.
Researchers at Cleveland Clinic discovered that pathogenic POLE/POLD1 genetic mutations in tumors lead to a high level of immune cell infiltration and improved response to immune checkpoint blockade therapy. The study's findings contribute to the growing list of discoveries that prove certain classes of drugs are more effective based o...
A study found that pharmacogenomic testing can help providers avoid prescribing antidepressants with undesirable outcomes. The test, which analyzes genes related to drug metabolism, resulted in a significant improvement in depression symptoms compared to usual care.
Researchers aim to develop a gene therapy that could suppress HIV replication and eliminate the need for life-long antiretroviral therapy. The study uses an AAV vector to produce leronlimab, a monoclonal antibody blocking HIV entry into immune cells.
A new gene therapy for sickle cell disease was tested in mice but proved ineffective due to the complex genetic make-up of the Berkeley mice. The study suggests that researchers should carefully consider the genetics of the mice used to test human diseases.
Experimental study finds large DNA insertions caused by retrotransposition can increase cancer risk in human cells edited with CRISPR/Cas9. In contrast, base editing and prime editing show much lower rates of retrotransposition.
A preliminary study published in eLife suggests that sperm screening can identify potentially harmful new genetic mutations and help prevent them from being passed on to offspring. The study found that 55 mutations were detected in the men's sperm, including 15 passed on to an embryo.
A new CRISPR strategy, employing natural DNA repair machinery, provides a foundation for novel gene therapy strategies to cure genetic diseases. The technique, known as homologous chromosome-templated repair, uses "nicks" of single DNA strands to correct genetic defects.
Researchers at Georgia Institute of Technology have developed a new screening technique called DNA barcoding, which accelerates the discovery of effective lipid nanoparticle carriers. The technique allows for simultaneous testing of many experiments and has improved nanoparticle pre-clinical screening.
A novel liquid biopsy test can accurately detect cancer DNA in the blood of patients with metastatic breast cancer within five hours, potentially helping oncologists determine if treatments are working. The test's diagnostic accuracy is 85%, correctly detecting cancer 83% of the time and ruling out cancer 92% of the time.
A team of researchers has developed an approach to minimize off-target mutations caused by the CRISPR-Cas9 gene-editing tool. The new method, dubbed spacer-nick, uses a modified pair of molecular scissors that make nicks on opposite strands of the DNA at two different points, reducing errors and increasing precision.
Researchers identified a three-gene signature in multiple myeloma tumors that predicts a positive response to selinexor-based therapy. The discovery could improve patient selection for targeted agents and expand the use of the drug into patients who haven't failed other therapies.
A new study from Tel Aviv University developed a unique treatment for AIDS that may be developed into a vaccine or a one time treatment. The treatment involves engineering type B white blood cells to secrete anti-HIV antibodies in response to the virus.
Researchers used stem-cell models to uncover hidden genetic markers of glaucoma, a leading cause of permanent blindness. The study identified 312 genetic variants associated with retinal cells and 97 genetic clusters linked to damage caused by glaucoma.
Researchers have identified three distinct brain circuits in the thalamus that contribute to Parkinson's disease symptoms, including motor dysfunction and depression. By manipulating these circuits, they were able to reverse Parkinson's symptoms in mice, suggesting potential new therapeutic targets.
A Rutgers study analyzing brain stem cells of autism patients found irregularities in early brain development, supporting the concept that ASD arises from poor control of brain cell proliferation. The study discovered that some patients had NPCs producing too many brain cells while others had underproduced cells.
Researchers have developed a gene therapy that appears to correct a rare creatine deficiency disorder by increasing creatine levels and reducing toxic guanidinoacetic acid. This could hold promise for treating the disorder, which commonly results in intellectual disabilities and seizures.
Researchers found that severe asthma patients produce growth factors that block corticosteroids from working, leading to frequent breathing problems. This discovery may lead to new treatments targeting these growth factors to improve outcomes for patients with severe asthma.
A new interactive web portal, SpUR, catalogues over 1,000 splicing events found in cancers, highlighting their role in tumor development and progression. The database provides a platform for researchers to study RNA dysregulations in cancer and develop RNA-based anti-cancer drugs.
Researchers used CRISPR technology to modify the ratio of amylose and amylopectin in potatoes, increasing culinary and industrial uses. The study found that potatoes with high amylopectin content have potential applications in bioplastics, food additives, adhesives, and ethanol production.
Two preclinical studies have identified potential new therapies for patients with Allan-Herndon-Dudley syndrome (AHDS), a brain development disorder that causes severe intellectual disability and movement problems. A gene therapy approach has shown promise in improving cognitive and motor functions, while repurposing a common drug may ...
Researchers at UNIGE discovered that temperature changes trigger organ-specific effects in all tissues, leading to distinct gene expression profiles. The study's web-based application provides access to thousands of gene expression profiles, enabling further research and potential therapeutic applications.
A new study reveals that individuals with sickle cell disease spend approximately $1.7 million on lifetime medical expenses, highlighting the significant economic burden of living with this disease. The study found that out-of-pocket costs for those with private insurance plans can be as high as 5-10% of their annual income.
Researchers found that people living with SCD have more medical appointments and urgent care visits, leading to higher out-of-pocket costs of $1,300 annually. The study suggests that curative therapies could reduce these costs, but accessing them will depend on conversations among policymakers, insurers, and patients.
Researchers have identified 10 new genes linked to hearing loss, casting doubt on the understanding that age-related hearing impairment originates mainly from sensory hair cells. The study suggests that the stria vascularis, a part of the cochlea in the ear, is a new target for treatments to help people with hearing loss.
A proof-of-concept study suggests that postnatal gene therapy may prevent or reverse deleterious effects of Pitt-Hopkins syndrome, a rare genetic disorder. The treatment restored normal activity of the deficient gene, preventing anxiety-like behavior and memory problems in an animal model.
A gene therapy approach has effectively reduced neuropathic pain in mice with spinal cord or peripheral nerve injuries without detectable side effects. The treatment, which targets impaired neurons, resulted in long-lasting benefits persisting for at least 2.5 months.
Researchers developed a droplet-based microfluidic technology to produce micro-organospheres from cancer patient biopsies within an hour. These miniature tumors retain the original microenvironment and can be used for testing many drug conditions, showing almost perfect correlation with actual clinical treatment outcomes.
Scientists have discovered a master gene that programs ear hair cells into either outer or inner ones, enabling the development of these cells to restore hearing. This breakthrough could provide a previously unavailable tool to create specific hair cells and improve treatments for age-related hearing loss.
Researchers used gene therapy to recover the TCF4 gene's function in human brain tissue, rescuing neural structure and function in brain organoids. The study offers promising insights into treating neurological disorders like autism spectrum disorders and schizophrenia.
José McFaline-Figueroa, a genomicist at Columbia University, has received a three-year NSF CAREER Award to investigate how cancer cells respond to anti-cancer therapy. His research aims to understand the molecular changes induced in aggressive cancer cells after exposure to treatment and how these changes alter response to treatment.