Articles tagged with Gene Delivery
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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.