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 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.
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.
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 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.
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.
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 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.
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.
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 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.
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.
Researchers found that genetic mutations in the MAPK pathway, key to normal cell growth, can also make head and neck cancer vulnerable. Individualized genomic analysis can identify specific mutations and target drugs, offering a promising approach to precision medicine.
The Lustgarten Foundation has awarded a $5 million grant to Break Through Cancer to target KRAS in pancreatic cancer. The Conquering KRAS in Pancreatic Cancer Team will integrate clinical and laboratory approaches to understand why patients respond to new therapies.
A new cell therapy has shown promising results in slowing the progression of Duchenne muscular dystrophy, a rare genetic disorder. The treatment uses heart cells to improve skeletal muscle and heart function, offering a potential cure for advanced disease cases.
Researchers load CAR-T cells with an oncolytic virus to target and kill solid cancer tumors, providing a potent immune response. The combination approach overcomes challenges in treating solid tumors with CAR-T cell therapy alone.
Researchers identified a signature of nonresponse to CAR T therapy in leukemia cells, characterized by DNA methylation and stem cell-like phenotypes. Decreased expression of genes involved in antigen presentation also hindered the immune response.
Scientists have developed a novel approach to targeting transcription factors, which could lead to new therapies for cancer and other diseases. A peptide designed to target the Mediator complex has been shown to selectively inhibit p53, a critical gene in human development and stress response.
A study found that 12% of recurrent ductal carcinoma in situ (DCIS) cases were new primary lesions unrelated to the original tumor. These findings suggest that genetic biomarkers for predicting recurrence may not be effective for all patients.
Researchers developed a MOF-based system for delivering DNA into target cells, overcoming challenges in gene therapy. The tiny structures protected genetic cargo and helped ferry it into the nucleus, where gene activity takes place.
Researchers identified a genetic variant associated with increased response to anti-PD-1 therapy and higher immune-related side effects in lung cancer patients. The variant, found in 15.7% of exceptional responders, may be used to identify patients who would benefit from treatment.
Researchers at the Babraham Institute have successfully developed a method to 'time jump' human skin cells by 30 years, restoring their specialized function. The new technique uses a partial reprogramming approach, allowing cells to retain their unique characteristics while still rejuvenating.
A new UCI-led study indicates base editing has tremendous potential to treat inherited retinal degeneration, particularly in Leber congenital amaurosis (LCA) patients. The treatment rescued cone photoreceptors on a long-term basis, suggesting a one-time, durable treatment for LCA patients.
Researchers found that SLN360, an experimental siRNA therapy, significantly reduced blood levels of lipoprotein(a), a lesser-known driver of heart disease risk. Participants who received higher doses saw their lipoprotein(a) levels drop by up to 98% and remained lower even after five months.
A gene therapy trial has shown significant reduction in infections and improvement in eczema symptoms in children with Wiskott-Aldrich syndrome (WAS). The treatment, using lentiviral gene therapy, offers a safe alternative to bone marrow transplantation for patients without suitable donors.
Researchers at Penn Medicine have developed a new approach to alter immune cells for CAR T cell therapy in just 24 hours, cutting manufacturing time from nine to 14 days. This could make the therapy more cost-effective and accessible to more patients.
Scientists have developed a new approach to expand the target range of CRISPR/Cas systems, allowing for slight variations in target DNA while maintaining local specificity. This technology could help realize the potential of CRISPR/Cas-based gene therapy and pathogen diagnosis, particularly for diagnostics.
Researchers have developed a gene therapy that restores night vision in dogs with congenital stationary night blindness (CSNB), a condition affecting the ON bipolar cells. The treatment enables dogs to navigate mazes in dim light and has a lasting therapeutic effect, paving the way for potential human treatments.
A new study led by Kelly Monaghan at West Virginia University suggests that interrupting the immune response may improve multiple sclerosis outcomes. The researchers found that targeting a specific protein called CCL17 can prevent the disease from attacking the central nervous system, leading to milder symptoms and delayed paralysis.
A gene called Fer2 has been identified as a key player in protecting dopaminergic neurons from degeneration. In flies and mice, overproduction of Fer2 reduces oxidative stress and prevents neurological defects characteristic of Parkinson's disease.
A clinical trial found that genomic sequencing enabled 107 patients to receive matched therapy, increasing treatment options for those experiencing cancer relapse. The study identified previously unknown mutations and showed promise for using circulating tumor DNA to identify targetable alterations.
Scientists have developed a genetic screening platform to identify genes that enhance immune cells' persistence and ability to eradicate tumor cells. By combining these genes with existing CAR-T cell therapy, researchers were able to engineer T cells that are more effective at eliminating tumor cells.
A novel surgical approach and synthetic viral vector Anc80L65 successfully delivered genetic cargo to the inner ear of nonhuman primates, paving the way for a potential treatment for hearing loss and vestibular disorders. The study's findings suggest that this method could be translated to humans.
Researchers have introduced a new mRNA delivery method to treat muscular dystrophy, which has already proven successful in mice. The method uses messenger RNA to correct genetic defects and has shown promise in clinical trials.
Researchers have genetically engineered a rat model of Down syndrome to test new therapies and explore the condition's unique genetics. The rats exhibit cognitive impairments, anxiety, and hyperactivity similar to humans with Down syndrome, providing a valuable tool for medical research.
Researchers discovered that an inorganic polyphosphate released by nerve cells contributes to the death of motor neurons in people with ALS and frontotemporal dementia. The study found that lowering levels of this toxin may be an innovative therapeutic strategy for diverse types of ALS/FTD.
Scientists developed a powerful new tool called Giraffe to improve genomic research by leveraging global genetic diversity. The tool allows for the use of a diverse pangenome reference point, enabling faster and more sensitive comparisons of short-read human genome sequences.
A clinical trial at UC Davis Health showed that cellular therapy offers promise for patients with late-stage Duchenne muscular dystrophy, stopping deterioration of upper limb and heart functions. The therapy appears to be safe and effective in improving skeletal muscle and cardiac function.
A new mathematical framework has been created to study fitness landscapes of regulatory DNA, enabling the prediction of gene expression changes. The framework uses a neural network model trained on millions of experimental measurements to decipher the evolutionary past and future of non-coding sequences.
Researchers uncover the pleiotropic functions of hnRNPK in regulating skeletal muscle cell differentiation, including inhibition of myoblast differentiation and suppression of genes involved in endoplasmic reticulum stress. The study suggests that targeting hnRNPK could be a potential therapeutic strategy for treating human disorders.
Scientists at the University of Texas at Austin have redesigned a key component of the widely used CRISPR-based gene-editing tool Cas9 to be thousands of times less likely to target the wrong stretch of DNA. The new version, called SuperFi-Cas9, is as efficient as the original but reduces off-target interactions, making it potentially ...
Researchers have discovered a genetic difference in the meniscus that makes about 50% of females more prone to developing knee osteoarthritis than males or other females. The study suggests potential for a blood test to identify high-risk individuals, allowing for early interventions and prevention strategies.
Researchers discover a genetic mechanism linking ALS and dementia to UNC13A protein corruption, providing hope for new treatments by blocking corrupted instructions. The study found that genetic variants increase the risk of UNC13A mRNA corruption in patients with ALS and FTD.
The five-year grant will be used to mentor junior scientists, leveraging City of Hope's expertise in cancer treatment and patient care. The program will provide students with hands-on experience in cell-based therapies, regulatory approval, and commercialization.
A Phase 3 clinical trial shows that gene therapy can end the need for monthly blood transfusions in children with transfusion-dependent thalassemia. The treatment uses a patient's own stem cells modified with a healthy hemoglobin gene, allowing patients to achieve transfusion-free status within months.
Scientists have developed a new therapy called CINDELA, which employs CRISPR-Cas9 to kill cancer cells while leaving normal tissues intact. The treatment targets specific mutations found in cancer cells and induces cell death through DNA double-strand breaks.