Scientists at Scripps Research Institute have developed a molecular switch that enables precise control of gene therapy doses. The technique involves embedding an RNA molecule called a hammerhead ribozyme into the genes used in gene therapies, allowing doctors to regulate the dosing level.
Scientists at Scripps Research developed a method to rapidly discover compounds that restore anti-tumor activity in immune cells called T cells. The approach identified 19 compounds that can reawaken exhausted T cells, potentially leading to new combination-immunotherapy regimens against cancers and persistent infections.
A Scripps Research study found that blocking the kappa opioid receptor while on a restricted-calorie diet resulted in significant weight reductions. Blocking this receptor may be a potential treatment approach for obesity.
Researchers clarify how RNA molecules fold during assembly of ribosomes, a complex process previously believed to be tightly controlled. The discovery opens up possibilities for designing targeted antibiotics with fewer side effects and better understanding of cancer growth.
Scientists at Scripps Research Institute have discovered a little-known protein that plays a crucial role in managing glucose and insulin levels in the body. The protein, PGRMC2, acts as a chaperone for heme, a molecule essential for cellular processes, and its absence is linked to obesity and metabolic diseases.
The experimental HIV vaccine developed by Scripps Research and IAVI successfully elicited broadly neutralizing antibodies to at least two vulnerable sites on HIV. The test results suggest that researchers are one step closer to developing an effective HIV vaccine, a major goal of medical science.
A new HIV vaccine strategy has been successfully demonstrated in proof-of-principle tests, targeting young immune cells and showing promise against the flu, dengue, malaria, and hepatitis C. The approach stimulates broadly neutralizing antibodies capable of neutralizing multiple strains of the virus.
Scripps Research scientists have discovered a way to stop a problematic enzyme from wreaking havoc on peripheral nervous system cells in patients with a specific subtype of Charcot-Marie-Tooth disease. By preventing the enzyme's entry into the nucleus, researchers achieved a complete rescue of symptoms in fruit fly models.
Researchers at Scripps Research have found an antibody that protects against a wide range of influenza viruses by targeting the neuraminidase protein. The discovery could lead to the development of a universal flu vaccine that offers broad protection against human, swine, and avian strains.
Researchers at Scripps Research identified cellular workings that stop and restart early brain development in tadpoles. When food is reintroduced after a period of starvation, neural progenitor cells resume dividing and the brain catches up on its growth.
Researchers at Scripps Research have discovered a key brain-cell change that may drive opioid addiction. By reversing this change, they hope to reduce symptoms of addiction and develop new treatments.
Scientists at Scripps Research Institute have developed an efficient method for synthesizing bilobalide, a plant compound with potent effects on insects but no toxicity to humans. The new synthesis allows easy access to related compounds for potential use as medicines and insecticides.
A plant-based compound closely related to resveratrol has been found to enhance gene delivery to hematopoietic stem cells, reducing antiviral restriction at endosomes. This breakthrough enables faster and more efficient treatments for diseases such as sickle cell anemia and leukemia.
A new genomics method, ANEVA-DOT, can identify disease-causing genes in patients with rare muscular dystrophies. The technique compares maternal and paternal allele activity levels across the genome to detect abnormalities.
Researchers discovered that cyclophospholipids can help protocells build stable vesicles, a crucial step in the emergence of life. This finding provides a solution to the puzzle of how stable vesicles could have developed in early Earth's harsh conditions.
Charcot-Marie-Tooth disease causes damage to the peripheral nervous system, affecting balance and motor skills. Researchers found that mutated enzymes take on an unusual shape, leading to unwanted interactions with nearby proteins and potential disease severity.
Scientists mixed RNA and DNA to study how life began on Earth, finding unstable chimeras that can replicate RNA and DNA. The research suggests a more complex system may have existed before pure RNA and DNA, supporting alternative theories of life's origins.
Scientists at Scripps Research Institute have identified brain cells that suppress drug cravings, which could lead to improved addiction treatments. The discovery sheds new light on how the brain responds to environmental cues that prevent relapse, providing hope for more effective medicines in the future.
Scientists at Scripps Research Institute have developed a new method for creating hindered ethers, which are integral to many drugs and commercial products. The new method uses electrochemistry and has improved production yield and reduced expense compared to traditional methods.
Researchers at Scripps Research Institute have discovered a potential biological marker for early detection of type 1 diabetes using single-cell analysis of immune cells. The discovery could lead to earlier intervention and prevention of the disease, which affects over 1.25 million Americans.
A genomics study reveals an undetected Zika outbreak in Cuba in 2017, which peaked around the same time international health agencies thought the epidemic was nearing its end. The study uses genomic sequencing and travel patterns to reconstruct virus ancestry and outbreak dynamics, providing insights into how viruses can spread silently.
A new study from Scripps Research provides unprecedented insights into how visual signals are distributed to different brain regions. Researchers discovered that similar proteins travel preferentially to one brain region, while some are transported to multiple regions.
A team of researchers at Scripps Research Institute has discovered an anti-opioid pathway that counteracts the effects of opioids on neuronal firing. The discovery could lead to the development of new pain medications with less potential for abuse and overdose.
Experts Eric Topol and Kai-Fu Li argue that international collaboration outweighs confrontation in the development of artificial intelligence for healthcare. They critique the forced divestiture of iCarbonX as misguided and highlight the need for access to clinical data to compete in AI health.
Researchers at Scripps Research Institute find that proteinaceous amino acids react more efficiently, leading to the formation of complex life forms. The study suggests a possible explanation for the selection of positively charged amino acids in proteins.
A new viral HIV vaccine has successfully protected rhesus macaques from high-dose SIVmac239 challenges, a notoriously difficult-to-defeat strain. The vaccine uses a gene-therapy inspired approach and durable protection is achieved with a single inoculation.
New research suggests that exosomes play a profound role in creating brain cells and circuits. The study found that exosomes can restore health to brain cells affected by developmental diseases, including Rett syndrome.
A seven-year NIH grant will support refinement and manufacturing of novel vaccine candidates designed to induce broadly neutralizing antibodies capable of disarming numerous HIV strains. The goal is to develop a vaccine that can halt the epidemic, despite current challenges posed by the virus's ability to rapidly evolve.
Researchers identified a key molecule, mir-135b-5p, found elevated in the brain's emotional memory processor that drives PTSD symptoms. Suppressing this molecule enables faster recovery from trauma, and it may also serve as a novel biomarker for treatment.
A Scripps Research study analyzed pharmacy insurance claims from over 35 million Americans and found that popular brand-name drugs experienced 'continual, marked, annual increases' between 2012 and 2017. The researchers concluded that the complex rebate system drives up costs for consumers.
Researchers discovered that Rqc2 marks protein fragments with a flag, allowing proteases to cut up bad fragments. The study sheds light on the ancient mechanism used by bacteria to recycle incomplete proteins, which has implications for understanding life's origins and developing new treatments for diseases.
A protein from Simian Immunodeficiency Virus (SIV) has shown promise as a potential component of an HIV vaccine, eliciting antibodies that neutralize infection against multiple HIV strains. The study uses SIV Env proteins to stimulate the immune system to produce protective antibodies.
A new study reveals that a previously unknown neural circuit, PPL2, plays a crucial role in regulating the strength of memories in fruit flies. The researchers found that activating this circuit can strengthen or weaken memories, depending on the context.
Scientists at Scripps Research have discovered that the Rhes protein creates tunnel-like nanotubes that enable the toxic Huntington's protein to travel between neurons, contributing to brain cell destruction and disease progression. This finding improves understanding of how Huntington's disease attacks certain brain cells.
Adult mouse models of SYNGAP1 disorder show improvement in measures of seizure and memory after gene repair, suggesting potential for treatment at any stage of life. The study offers a path to measure the effectiveness of potential medications or therapies.
Scientists at Scripps Research have identified small molecules that prevent structural changes to proteins causing the disease. These molecules bind to and stabilize immunoglobulin light chain proteins, preventing misfolding and forming toxic plaques.
Researchers at Scripps Research have developed a potential drug that targets the genetic defect causing myotonic dystrophy type 1, a disease affecting 1 in 2,500 people. The therapy, called Cugamycin, improves muscle defects without harming healthy gene transcripts.
Researchers have discovered a compound that can convert RNA building blocks into DNA building blocks, challenging the popular 'RNA World' hypothesis. This finding suggests that early life forms may have used both RNA and DNA, contrary to the prevailing view that they arose separately.
Scientists at Scripps Research discovered a life-extension pathway in C. elegans worms that modulates a cannabinoid biological pathway, extending lifespan by up to 45%. The study introduces a powerful method for applying chemical probes to lab animals like worms to discover biology relevant to humans.
Researchers at Scripps Research have discovered a specific neuronal population responsible for alcohol cravings, using lasers to temporarily inactivate these cells. This breakthrough may lead to the development of drug therapies or gene therapies for alcohol addiction.
Researchers at Scripps Research Institute have identified a promising small molecule that targets the HA stem of influenza A group 1 viruses, demonstrating effectiveness against seasonal and pandemic strains in mice. The orally active compound, JNJ-4796, has the potential to be used as a treatment for flu sickness.
Researchers at Scripps Research Institute create a battery-like system to manufacture medicines, avoiding safety risks and increasing versatility. They use additives from lithium-ion battery manufacturing to conduct reductive electrochemistry safely and efficiently.
Researchers at Scripps Research Institute have developed a new approach to shifting breast cancer cells into HER2-positive status using RNA-targeting precision medicines. The proof-of-concept study suggests that this technology could expand treatment options for patients with dwindling choices.
Matthew D. Disney's laboratory has proven that RNA can be a small-molecule drug target, offering new ways to tackle incurable diseases. His work has already provided lead medicines for genetic diseases and difficult-to-treat cancers.
Scientists at Scripps Research have discovered how neurons manage mitochondrial transport, a process crucial for nerve cell function and energy production. The study found that cAMP signaling enhances mitochondrial transport after synapse formation, requiring significant energy to maintain communication between cells.
Researchers at Scripps Research Institute have identified an important intermediate step in how alcohol intoxication occurs. The enzyme phospholipase D2 links ethanol molecules to lipid membranes, triggering a metabolite called phosphatidylethanol that causes nerves to fire more easily, leading to hyperactivity in flies.
Researchers have discovered that protein clumps, particularly pα-syn*, recruit enzymes and tau to damage brain cells, leading to mitochondrial destruction. This study provides insight into the molecular mechanisms of Parkinson's disease, shedding light on potential treatment targets.
Scientists have discovered a molecular cause of rare autoimmune disorders, including Singleton-Merten syndrome and Aicardi-Goutières syndrome. Mistakes in RNA proofreading lead to improper interferon signaling, triggering autoimmunity.
A new RNA-binding compound has been found to block the most common genetic cause of both familial ALS and frontotemporal dementia, according to researchers at Scripps Research. The compound targets a specific form of RNA involved in protein production, potentially providing a new avenue for treating these diseases.
A new study published in Immunity shows that an experimental HIV vaccine protects rhesus macaques from infection by inducing neutralizing antibodies against a Tier 2 virus. The vaccine strategy, developed over two decades, aims to train the immune system to recognize and attack vulnerable areas on the HIV envelope protein.