Articles tagged with Gene Editing
Researchers used ex vivo lentiviral gene therapy to treat MPS IVA in mice, achieving partial correction of bone pathology and complete correction of heart pathology. The study suggests potential for novel therapies to treat patients with MPS IVA.
A CRISPR-Cas9 based gene editing therapy has been shown to reduce angioedema attacks and sustainably lower kallikrein levels in patients with hereditary angioedema, offering a potential treatment option. The therapy was tested on 27 patients across two dosages compared to placebo.
A USC Stem Cell mouse study identifies a small subset of blood stem cells as the primary driver of immune aging. The researchers found that this subset overproduces innate immune cells, leading to an age-associated imbalance and increased disease risk. By targeting this subset, the study suggests a potential therapy to delay immune agi...
Dr. Fyodor Urnav proposes a set of initiatives to address the crisis, including pooling patients by syndrome and permitting multiple gene editors in a single Investigational New Drug application. This approach aims to accelerate the development of CRISPR therapies for rare genetic diseases.
Researchers at ChristianaCare Gene Editing Institute use CRISPR tools to safely disable gene mutation linked to treatment-resistant melanoma. The approach targets melanoma tumor cells while leaving healthy cells alone, restoring sensitivity to anticancer drugs.
Researchers at Lehigh University are developing predictive models for gene editing with CRISPR to improve outcomes and expand medical applications. The team is using AI and advanced computer models to simulate the effects of altering a single gene on the entire genome, enabling them to predict and avoid unintended consequences.
A research team developed an RNA-based sensor platform that can regulate gene expression in bacteria, mimicking natural biological interactions. The START platform enables tunable control over sensor response and detection of various molecules, including drugs and proteins.
Researchers developed a compact 'gene scissor' tool, TnpB, which shows a 4.4-fold increase in efficiency of modifying DNA, making it more effective as a gene editing tool. The tool can be used to treat patients with familial hypercholesterolemia, reducing cholesterol levels by nearly 80%.
Ling Li's groundbreaking research improves global nutrition and sustainability by increasing protein content in rice and soybean crops. The gene-edited approach boosts nutritional value while promoting sustainable agricultural practices.
CAR-T cell therapy, a regenerative immunotherapy, has shown promise in treating blood cancers but struggles with T-cell exhaustion. Researchers have discovered that overproduction of the IL-4 protein causes this exhaustion and used CRISPR gene-editing technology to remove it, improving CAR-T cell therapy outcomes.
Researchers have shown that repeated administration of lipid nanoparticle-encapsulated mRNA therapy significantly extended survival and reduced serum leucine levels in a mouse model of maple syrup urine disease. The treatment approach may represent a potential long-term universal treatment for MSUD.
Researchers at the University of Hawaii have developed a new gene editing technology that can efficiently deliver healthy genes to the body. This method addresses limitations of current methods and has shown success rates of up to 96%, potentially leading to faster and more affordable treatments for various genetic diseases.
Researchers are exploring the use of CRISPR Prime Editing to optimize CAR-T cells for treating multiple myeloma and other cancers. The goal is to improve effectiveness, duration, and cost-effectiveness of CAR-T cell therapies.
Researchers used gene editing to restore hearing in adult mice with a form of inherited deafness called autosomal dominant deafness-50 (DFNA50). The approach involved shutting down a damaged copy of the microRNA (miRNA) gene, which enabled the animals to regain hearing. This method may eventually lead to potential treatments for inheri...
Researchers at Broad Institute of MIT and Harvard have developed a gene-editing approach that efficiently corrects the most common mutation causing cystic fibrosis, found in 85% of patients. The new method precisely and durably corrects the mutation in human lung cells, restoring cell function to levels similar to Trikafta.
Researchers developed a CRISPR/Cas9 genome editing approach to target and disrupt a specific mutation causing progressive hearing loss. The study found robust preservation of auditory function, with earlier intervention proving most optimal.
A team of scientists at Gladstone Institutes has developed a new method that enables them to make precise edits in multiple locations within a cell—all at once. They created a tool using molecules called retrons to efficiently modify DNA in bacteria, yeast, and human cells.
Researchers, including Assistant Professor James Lewis, are studying the evolution of butterfly wing color patterns as a model for understanding population adaptation to environmental changes. The study aims to understand why some Heliconiine butterflies lose their mimicry phenotype and how this affects their survival.
Researchers at the University of Sydney have developed SeekRNA, a programmable tool that can precisely target and relocate genetic sequences with high accuracy and flexibility. This breakthrough technology surpasses current limitations of CRISPR, enabling more precise editing and reducing errors.
New research reveals that CRISPR/Cas9 gene editing tools have biases against cells from people of African ancestry, leading to false negative results. The study's findings highlight the importance of increasing genetic diversity in large-scale cell line libraries to mitigate this bias.
Researchers developed an ex vivo gene-editing protocol to treat hereditary tyrosinemia type 1 using liver cell therapy. The study showed robust engraftment and expansion of transplanted gene-edited hepatocytes in recipient mice, correcting disease indicators such as normalized tyrosine, phenylalanine levels.
Scientists have successfully edited genes in a highly resilient tardigrade species using CRISPR technology, allowing them to investigate the genetic basis of its exceptional abilities. The research could lead to new insights into human organ preservation and transplantation.
Scientists have improved a gene-editing technology that can insert or substitute entire genes in the genome, potentially treating multiple genetic diseases with a single therapy. The new approach, eePASSIGE, uses prime editors and recombinase enzymes to make gene-sized edits several times more efficiently than previous methods.
Researchers found that Orco protein is necessary for the development and life of olfactory cells in ants. Mutant ants lacking Orco experience massive olfactory neuron loss, suggesting a link between Orco and neuronal survival.
Researchers have developed a compact, high-fidelity version of the Cas12a protein, which can be packaged within a non-pathogenic virus for targeted gene editing. The modified protein demonstrates efficient editing activity and has been shown to reduce blood cholesterol levels in mice with high cholesterol.
Researchers at Rice University have identified genes critical for enteric nervous system development using zebrafish embryos. Opioid signaling pathways are required for the formation of nerves in the gut, contradicting previous assumptions about their role in pain perception and addiction.
Researchers have developed a new gene-editing system called multiplexed orthogonal base editors (MOBEs) to tackle complex diseases. MOBEs enable simultaneous installation of multiple point mutations across the genome, reducing the risks associated with traditional gene-editing tools like CRISPR-Cas9.
A KAUST team developed a simple approach to tackle CRISPR's deletion issue by targeting error-prone DNA repair pathways. By modulating specific genes, they reduced large deletions while enhancing homology-directed repair efficiency.
Researchers at Fred Hutch Cancer Center have made progress in laboratory studies using gene editing to eliminate herpes virus infections. The experimental therapy eliminated 90% of HSV-1 after facial infection and 97% after genital infection, reducing viral shedding.
A pioneering CRISPR gene editing trial has demonstrated significant improvement in vision for 79% of participants with inherited retinal degeneration. The study's findings support further research into the potential of CRISPR-based treatments for inherited blindness.
A phase 1/2 trial of CRISPR gene editing has demonstrated safety and efficacy, with measurable improvements in 11 out of 14 participants with a form of inherited blindness. The treatment, EDIT-101, was found to be clinically meaningful for four participants and showed significant improvements in cone-mediated vision.
Researchers used CRISPR-Cas9 to genetically modify kissing bugs, opening a door to controlling Chagas disease. The new method, ReMOT Control, allows efficient and targeted editing of genes in eggs.
Researchers identified a small RNA-binding protein called La that promotes gene editing with high efficiency. The team created a new protein, PE7, which harnesses La's activity to enhance prime editing, leaving unwanted byproducts at low frequencies.
Researchers have shown that a single normal copy of a defective gene can prevent cysts in polycystic kidney disease. Additionally, they found that a type of drug called a glycoside can sidestep the effects of the defective gene and prevent cyst formation.
Researchers developed an Integrated Classifier Pipeline (ICP) tool to analyze CRISPR edit outcomes and track unintended 'bystander' edits. The ICP system provides a genetic fingerprint of how material is being inherited, helping scientists untangle complex biological issues.
Researchers successfully demonstrate CRISPR-Cas gene editing technology to eliminate all traces of the HIV virus from infected cells in laboratory settings. The study aims to develop a robust and safe combinatorial regimen to target diverse HIV strains across various cellular contexts.
Researchers have developed a new CRISPR-based approach to study the function of immune genes, bypassing challenges of modifying immune cells. By targeting stem cells that produce immune cells, scientists can knock out specific genes in different cell types at various times, providing greater insight into gene interactions and their rol...
Researchers created novel gene editing enzymes with improved precision, reducing off-target RNA edits by over 99%. The technology has potential applications in treating mitochondrial genetic diseases and may lead to transformative treatments within the next five years.
A Husker research duo has won a $25,000 prize in the NIH’s Targeted Genome Editor Delivery Challenge. They will advance development of universal milk exosomes capable of transporting gene editors to any location in the body, overcoming a significant challenge in using gene editing to treat disease.
Researchers developed a faster test for mpox using CRISPR and nanopore sensing technology, which can detect the virus in 32-55 minutes, compared to hours of current lab testing. The test is specific to mpox and was confirmed not to detect other viruses.
A breakthrough gene-editing therapy has been successfully treated over ten patients with hereditary angioedema, reducing symptoms by up to 95% and potentially providing a permanent cure. The therapy targets the KLKB1 gene responsible for producing plasma prekallikrein.
A new CRISPR delivery method enables precise targeting of specific cell subsets in living animals, paving the way for programmable gene therapy. The system uses antibody-targeted 'enveloped delivery vehicles' to selectively edit T-cells and create CAR T-cells.
Researchers used CRISPR to modify a tomato gene, resulting in reduced water consumption without affecting crop quality. The discovery holds implications for basic scientific knowledge and could help increase plant yields in dry conditions.
Researchers have developed CRISPR off-switches to mitigate off-target effects, a major concern in genome editing. The new technology, based on anti-CRISPR proteins, can block CRISPR-Cas3 machine function and prevent unintended edits.
A UC Riverside research project will focus on changes in the gene network that specify early intestinal precursor cells in nematodes like Pristionchus pacificus. Gene networks describe how genes turn each other on and off, and changes in these networks can lead to diseases such as cancer.
Researchers develop a delivery system for prime editing, enabling the correction of disease-causing genetic mutations in animal models. By adapting virus-like particles to carry the machinery for prime editing, scientists have achieved partial restoration of vision in mice with two genetic disorders.
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.
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.
Scientists at Florida State University produced the first high-resolution images showing magnesium ions playing a crucial role in CRISPR-Cas9's DNA-cutting process. The discovery sheds light on how magnesium coordinates double-stranded breaks, providing new insights into the enzyme's functioning.
Researchers improved biomass-related traits in sheepgrass using a custom genome editing system, revealing its potential for rapid genomic breeding. The study increased understanding of sheepgrass genomics and established a precedent for its genetic improvement.
Researchers employed heat-induced FT overexpression to accelerate assessment of floral phenotypes after CRISPR knockout of LEAFY and AGAMOUS genes in poplar trees. The study successfully induced early flowering and demonstrated a wide range of inflorescence and floral forms.
Researchers have identified a new mechanism by which phages evade CRISPR-Cas immune systems in bacteria, revealing a potential approach to make gene editing safer and more efficient. This discovery could lead to the development of bespoke anti-CRISPRs to neutralize CRISPR-Cas systems and provide an alternative to antibiotics.
A new CRISPR-based gene-editing tool, AsCas12f, has been developed with enhanced editing ability and compact size. The engineered enzyme has already shown success in animal trials and holds promise for improved treatments of genetic disorders.
Researchers create adenine base editor with 'on/off' switch, reducing off-target edits by over 70% and increasing accuracy of on-target edits. The tool has potential to correct nearly half of disease-causing point mutations in human genome.
A CRISPR-Cas3 system has restored dystrophin protein function in induced pluripotent stem cells from patients with Duchenne muscular dystrophy. The approach uses a dual CRISPR RNA method to remove large sections of the dystrophin gene, yielding truncated but still functional proteins for various mutation patterns.
Researchers used base editors to introduce specific combinations of activating and inactivating mutations into healthy organoids, creating realistic models for various types of cancer. This allows for further investigation into the development and treatment of cancer, with potential applications including testing new drugs.
A novel CRISPR-based gene-editing treatment, EBT-001, effectively removes SIV from the genomes of non-human primates without off-target effects. The study's findings support the development of a cure for HIV/AIDS in humans and pave the way for ongoing clinical trials.
City of Hope will conduct Phase 1 clinical trials for novel cell and gene therapy treatments for patients with HIV, acute myeloid leukemia, and severe aplastic anemia. A CAR T therapy for HIV is also being developed to potentially target other diseases.
Researchers at the Marine Biological Laboratory have devised a method to precisely alter rotifer genomes using CRISPR-Cas9, enabling the study of fundamental biology and evolution. The new approach will allow scientists to investigate various aspects of biology, including aging, DNA repair mechanisms, and mitochondrial function.
Researchers at the University of Arkansas successfully used gene editing to reduce 'chalkiness' in rice, a condition caused by heat stress. The modified rice lines showed a significant improvement in grain quality, with increased starch granule compactness and reduced chalkiness.