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 study by Sanford Burnham Prebys found that misfolded proteins in liver cells contribute to liver cancer development, potentially increasing the risk of certain patients receiving gene therapies for hemophilia. Improving FVIII folding could decrease this risk and develop safer treatments.
Researchers have discovered that a specific mutation in the misfolding protein causing Parkinson's disease can also protect against multiple system atrophy (MSA), another fatal neurodegenerative disorder. The findings provide a promising lead for developing targeted treatments using personalized medicine approaches.
Researchers have developed an AAV-based gene therapy to treat glutaric aciduria type I (GA-I), a rare genetic disorder causing accumulation of glutaric acid in the central nervous system. The treatment has shown promising results, with a survival rate of 100% in mice treated intracerebroventricularly.
Researchers at University of Pittsburgh have designed novel nanoparticles that co-deliver a chemotherapy drug and a novel immunotherapy, shrinking tumors in mouse models of colon and pancreatic cancer. The therapy silences a gene involved in immunosuppression by blocking Xkr8 protein distribution on the cell membrane.
Nucleic acid therapies aim to treat genetic disorders and diseases, but delivering therapeutics is a significant challenge. Researchers are investigating nanoparticle delivery systems to target specific cells and sub-cellular compartments for effective delivery.
Researchers have engineered a family of adeno-associated viral vectors that can deliver cargo to the primate brain, offering a safer and more efficient way to treat genetic diseases. The PAL family of AAVs has been shown to be three times better at delivering their cargo into the brain than current leading AAV delivery vehicle AAV9.
The UC San Diego Alpha Stem Cell Clinic will receive $8 million in funding to launch new clinical trials and improve accessibility of stem cell therapies. The clinic has already launched 59 clinical trials and treated 277 patients with various diseases.
The USC+CHLA Alpha Clinic will advance clinical trials of new cell and gene therapies, engaging underserved communities and training the workforce that conducts them. This program complements existing efforts to translate discoveries into new therapies for better health outcomes.
Researchers found that IGF1 gene therapy increases kisspeptin expression and GnRH release, and alters microglial cell numbers, suggesting a potential protective effect against reproductive decline. This could lead to new strategies for optimizing lifespan and combating age-related health problems in women.
A Rice University bioengineer has developed a noninvasive technology to measure gene expression in deep tissues, particularly in the brain. This innovation could improve the monitoring of gene therapy treating neurodegenerative disorders such as epilepsy, ALS, and Huntington's disease.
Researchers have developed a new gene therapy that selectively targets overactive brain cells, reducing excitability and suppressing seizures in mice. The treatment shows promise for treating neurological disorders such as epilepsy, Parkinson's disease, schizophrenia, and pain disorders.
Amanda Woerman's research aims to disrupt tau misfolding, a common thread in fatal neurodegenerative disorders PSP and CBD. The grant-funded study seeks to test a proof-of-concept gene therapy for these conditions, potentially leading to personalized medicine breakthroughs.
The Alpha Stem Cell Clinic will develop preclinical studies into early and later phase clinical trials with the goal of establishing advanced regenerative medicine treatments. The clinic will also foster greater collaboration with eight similar clinics across the state and educate the public about stem cell and related therapies.
Johns Hopkins Medicine researchers have developed a novel genetic engineering approach to deliver gene therapy by utilizing a cell's natural process to
A new Northwestern Medicine study identifies common and rare gene mutations that impact radiation resistance and sensitivity. This information will allow clinicians to better calibrate radiation doses based on genetic mutations, improving treatment efficacy while reducing toxicity.
Researchers at the University of Pennsylvania School of Medicine have developed a gene therapy that rapidly restores night vision in adults with LCA, a common form of congenital blindness. The treatment has been shown to improve rod-based visual functions and correct severe visual deficits in patients.
Nanoscope Technologies received a $1.5M NIH SBIR grant to develop gene-agnostic glaucoma therapy utilizing Engineered Mechanosensitive Channels (EMCs). EMCs can autonomously regulate pressure, offering a novel approach to treating Glaucoma.
Researchers at CNIO have identified alveolar type II pneumocytes as the primary cell type responsible for developing pulmonary fibrosis. The study reveals that targeting these cells through telomere-based therapy may lead to a breakthrough in treating this debilitating disease.
Researchers have used single-cell sequencing to uncover novel gene expression patterns in injured kidney cells, providing new avenues for biomarker discovery and treatment. The studies reveal that epithelial cells of all tubule segments are involved in the injury processes, with distinct molecular patterns across patients.
Researchers at Universidad de Navarra identified a biomarker that predicts CAR T cell therapeutic capacity, which could improve treatment outcomes for patients. The study found that high CAR density in CAR T cells is associated with a worse clinical response in hematological tumors.
Researchers developed a computational platform to identify metabolic vulnerabilities in ovarian cancer genes, suggesting opportunities for targeted therapies. The study found that certain genetic alterations can create vulnerabilities in cancer cell metabolism, which can be exploited to selectively kill cancer cells.
Researchers developed new educational materials to help individuals with sickle cell disease and their support networks learn about gene therapy clinical trials, benefits, risks, and treatment options. The project aims to facilitate engagement between the community and those conducting relevant clinical trials.
Scientists at St. Jude Children's Research Hospital have created a tool to identify 'safe harbor' sites for gene therapies, balancing safety and therapeutic expression. The tool uses genomic and epigenetic information to find regions with high variability among healthy individuals, reducing the risk of cancer or other problems.
Researchers discovered that autophagy facilitates the elimination of cancer cells via cell competition, highlighting its potential as a target for cancer prevention and treatment. The study sheds light on the role of autophagy in maintaining tissue homeostasis and opening avenues for novel anti-cancer therapeutics.
Researchers at UT Southwestern have identified a gene therapy target for polycystic kidney disease (PKD), which could lead to the arrest or cure of the condition. By blocking the inhibition of PKD1 and PKD2 gene expression, the researchers found that kidney cyst growth was hindered in ADPKD models.
New research estimates the overall disease burden of genetic risk factors on healthy life years lost, prioritizing interventions using genetic information. The study found that common genetic variants for cardio-vascular diseases and Alzheimer’s disease have a substantial population-level impact.
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.
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 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.
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.
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 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.
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 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.
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...
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.