Articles tagged with Gene Delivery
In vivo CAR-T therapy reduces manufacturing time and cost, enabling more accessible cancer immunotherapy. The strategy also enables superior self-renewal capacity and anti-tumor persistence.
A novel autologous gene therapy for Wiskott-Aldrich syndrome has demonstrated durable clinical benefits in a phase 3 study, with 96% survival at 1 and 5 years. The treatment, etu-cel, has also reduced severe infections and moderate-to-severe bleeding events.
Researchers at Nagoya University developed a new delivery vehicle for circular RNA (cirRNA) using a novel lipid nanoparticle, FL0445-LNP, which improves the stability and efficacy of mRNA-based therapeutics. The technology has potential applications in cancer vaccines, genome editing, and protein supplements.
Researchers developed a method to inhibit lipid nanoparticle migration into the liver by coating hepatic sinusoidal walls with polyethylene glycol. This coating reduced liver accumulation by several dozen times and increased protein expression in the spleen, promoting safer nanomedicine with lower dosages.
Researchers have successfully engineered plants to produce myoglobin, an important component of animal muscle, using a gene gun to insert the genes into chloroplasts. The yield was approximately three times higher than when inserted into the nuclear genome, paving the way for plant-grown meat production.
Researchers at UMass Chan Medical School have developed a microRNA-based gene therapy that suppresses mutant SOD1 production, delaying disease onset by 60 days and extending lifespan by 100 days in mice models of ALS. The therapy, delivered via adeno-associated virus (AAV) vector, preserves motor neurons and maintains neuromuscular con...
A gene therapy developed at UAB prolongs health span and preserves the function of multiple organs in mice during aging, with sustained beneficial effects across multiple endpoints. The treatment shows improved metabolism and organ function, including weight management, insulin sensitivity, and increased energy expenditure.
Philip Tai, PhD, received a $1.6 million grant to investigate AAV vector mechanisms using high-resolution DNA sequencing technology. His findings could lead to new vector designs that improve gene therapy treatments' safety. The goal is to remove mutations that cause cancer-causing integration into host cells.
Researchers have developed a new gene editing technology called 'prime assembly' that allows efficient insertion of large DNA segments into the human genome. This innovation enables treatment of genetic diseases by replacing entire genes, promising substantial progress in treating conditions with multiple mutations.
A new study reveals that bacteria can actively limit the spread of antibiotic resistance genes by deploying molecular gatekeepers called YokF. This mechanism blocks the transfer of beneficial genes, giving microbes a competitive advantage in dense microbial communities.
Oregon State University researchers have developed a new class of lipid nanoparticles that can safely deliver gene-editing tools at lower doses, resolving a longstanding challenge in the field. The breakthrough was made possible by a DNA-based barcoding test that measures how efficiently different nanoparticle designs release their cargo.
Genethon's GNT0004 gene therapy shows long-term efficacy in patients with Duchenne muscular dystrophy, maintaining clinical efficacy and safety at two years. The trial included 72 boys aged 6-10 with retained walking ability, treated with GNT0004 at a therapeutic dose.
Pompe disease is a rare genetic disorder caused by a deficiency in the GAA enzyme, leading to glycogen accumulation in cells. Genethon's gene therapy approach has shown preclinical efficacy in animal studies, correcting glycogen accumulation and improving cardiac hypertrophy and muscular dysfunction.
Waskyra, an ex vivo gene therapy, offers new hope for patients with Wiskott-Aldrich syndrome by reducing severe bleeding events and serious infections. The FDA approval confirms Fondazione Telethon's excellence in rare genetic disease research.
Fondazione Telethon's gene therapy Waskyra, treating Wiskott-Aldrich Syndrome, has received CHMP positive opinion and marketing authorisation in the European Union. The therapy offers new hope for patients affected by this rare genetic disorder.
A nonsurgical approach has been demonstrated to quiet a specific brain circuit in an animal model by delivering engineered gene therapy only to the targeted region. The method uses low-intensity focused ultrasound to open the blood-brain barrier, allowing precise control over brain activity without impacting off-target areas.
A naturally occurring gene called Cyclin A2, normally silenced in humans, can make new functioning heart cells and aid in the heart's repair. The breakthrough discovery could lead to new techniques for repairing damaged hearts as an alternative to transplants or implanted cardiac devices.
Macromolecular gene delivery systems are advancing non-viral therapeutics by overcoming challenges like lower transfection efficiency and stability issues. Innovations in polymer design, functionalization, and targeting mechanisms are paving the way for clinically viable non-viral treatments.
A Phase I/II clinical trial found that gene therapy reduced seizures, improved oral feeding, and increased production of the HexA enzyme. Participants experienced fewer and more controllable seizures, and some remained on full oral feeds for up to 27 months.
A new study shows that delivering a single injection of gene therapy at birth may offer years-long protection against HIV. The treatment uses an adeno-associated virus to deliver instructions to muscle cells, which produce broadly neutralizing antibodies capable of neutralizing multiple strains of HIV.
Genethon is launching a Phase 3 clinical trial in Europe for its low-dose microdystrophin gene therapy GNT0004, targeting boys aged 6 to 10 with retained walking ability. The trial aims to demonstrate efficacy and tolerance of the treatment.
Gene therapy relies on efficient and safe delivery of therapeutic genes to target cells. Macromolecular carriers, including synthetic and natural polymers, offer biocompatibility, controlled release, and targeted delivery. These systems have shown promise in treating genetic disorders and complex diseases like cancer.
Researchers at the University of Sydney developed a biological 'artificial intelligence' system called PROTEUS, which can accelerate cycles of evolution and natural selection to create molecules with new functions in weeks. The system has potential applications in finding new medicines and improving gene editing technology like CRISPR.
Researchers at Iowa State University developed a new internal barrel design for the 'gene gun' technology, improving its efficiency by up to 50 times. The Flow Guiding Barrel reduces particle loss and uneven distribution, enabling more consistent and reliable genetic material delivery into plant cells.
A new gene therapy delivery device called NANOSPRESSO could revolutionize how hospitals treat rare diseases by allowing them to create personalized nanomedicines in-house. This democratized approach to precision medicine could boost access to low-cost bespoke gene and RNA therapies, especially in low-resource settings.
Researchers developed nanomachines that can efficiently deliver antisense oligonucleotides to sentinel lymph nodes, reducing TGF-β1 levels and reactivating depleted CD8-positive T cells. This enhances cancer treatment outcomes for advanced breast cancers with no effective treatments.
Researchers developed a gene therapy that can target the airway and lungs using a nasal spray, outperforming previous versions in preclinical models. The innovative tool, AAV.CPP.16, showed promise for treating respiratory diseases like pulmonary fibrosis and viral infections.
Researchers successfully created over 1000 new biological tools, known as enhancer AAV vectors, that can target specific brain cell types. These tools offer unprecedented access to brain cells and hold promise for targeted gene therapies to correct genetic defects in specific cells contributing to disease.
Scientists have uncovered a previously unknown mechanism explaining how neurons survive botulinum neurotoxin type A exposure. The research found that specific tRNA fragments interact with key proteins and RNA molecules involved in regulating ferroptosis, supporting neuronal survival by blocking cell death pathways.
Researchers from Mass General Brigham presented key findings from multiple innovative studies on gene and cell therapy, focusing on rare diseases, brain cancer, and neurodegenerative disorders. The studies explored strategies to improve care delivery and accelerate translation from lab to clinic, with potential breakthroughs in treatin...
Researchers at UCSF are enrolling patients in a clinical trial to correct the genetic mutation causing sickle cell disease using non-viral CRISPR-Cas9 gene editing. The therapy aims to eliminate the need for a bone marrow transplant and create a new blood system free of the disease.
Researchers developed a simple and efficient method for diversifying reactive end-groups on poly(2-oxazoline)s, enabling rapid exploration of poly(2-oxazoline)-based nanomedicine platforms. The approach was shown to produce POx-based lipid nanoparticles comparable in transfection capability to their PEGylated counterparts.
Researchers from Kyushu University and Harvard Medical School have identified proteins that can reprogram fibroblasts into cells with properties similar to limb progenitor cells. The new method simplifies the process of regenerating human limbs after amputation and could one day be used to give snakes back their legs.
Rice University bioengineers Jerzy Szablowski and Julea Vlassakis have received the National Institutes of Health Director’s New Innovator Award for their creative research projects on gene expression and cancer interactions. Szablowski is developing noninvasive methods to map gene expression, while Vlassakis is studying complex single...
CHOP and Penn Medicine researchers have developed a proof-of-concept model for delivering gene editing tools directly into diseased blood cells within the body. This approach aims to reduce costs and increase access to gene therapies for blood disorders, which currently require chemotherapy and stem cell transplants.
Scientists have developed a new method to deliver genetic information to stem cells using nanoparticles coated with a specific polymer, enabling more efficient control over cellular differentiation. This innovation has the potential to improve the efficiency and effectiveness of regenerative medicine treatments.
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.
A UMass Chan clinical trial demonstrates the safety and efficacy of an antisense oligonucleotide in suppressing mutant C9ORF72, a common cause of familial ALS. The treatment led to reduced levels of neurotoxins and stable or improved ALS functional scores.
Researchers at UC Berkeley have developed a new CRISPR editing technology that enables simultaneous editing of genes in multiple cell types and species within a microbial community. This approach, called community editing, has the potential to track edited microbes and understand their functions within complex ecosystems.
Nanoscope Technologies is featuring groundbreaking research on optical gene delivery and functional characterization platform technologies for treating retinal diseases. The company's lead product, vMCO-010, delivers light-sensitive Multi-Characteristics Opsin into retinal cells to restore vision in patients with degeneration.
Nanoscope Technologies has been awarded a National Institutes of Health (NIH) Audacious Goal Initiative grant to develop ultrafast laser-based gene delivery technology for vision restoration. The company aims to re-sensitize photo-degenerated retinal areas with a highly photosensitive Multi-Characteristics Opsin (MCO) using an infrared...
Researchers found that transient activation of the Hedgehog pathway after irradiation rescued salivary gland dysfunction by preserving stem/progenitor cells and parasympathetic innervation. Shh gene transfer improved saliva flow rates in mice, while also preserving acini marker Aqp5 expression.
Researchers have developed a new gene delivery method using magnetically targeted nanoparticles that can effectively deliver genes to injured arteries without causing side effects. The technique, which uses stents as a platform for magnetic guidance, shows promise in overcoming current limitations of gene therapy vectors.
Researchers at Northwestern University developed a novel gene delivery technology using nanodiamonds, achieving 70 times greater delivery efficiency than conventional methods. The surface-modified particles successfully delivered DNA into mammalian cells while preserving biocompatibility.
Researchers developed gene therapy targeting phospholamban, a protein contributing to heart failure. Studies in rats showed improved heart function and reduced scar tissue formation after treatment.