摘要:
An apparatus, method, and use for ultrasound mediated microbubble delivery of pharmaceutical compositions to pulmonary tissue are provided. The pulmonary ultrasound apparatus includes an ultrasound signal generator for generating ultrasonic signals, an ultrasound transducer assembly having an ultrasound transducer operatively connected to the ultrasound signal generator, the ultrasound transducer configured to transmit the ultrasound signal generated by the ultrasound signal generator to pulmonary tissue, wherein the ultrasonic signal is transmitted at a frequency, a pressure, and a pulse duration for cavitating microbubbles to deliver a pharmaceutically active molecule to the pulmonary tissue.
摘要:
A method to prevent, inhibit or treat one or more neurological symptoms associated with a central nervous system disorder, e.g. MPSI or MPSII by, for example, intrathecally, intracerebroventricularly or intravenously administering a rAAV encoding a gene product associated with the disease, e.g., administering to an adult mammal in which the gene product is absent, defective or present at a reduced level relative to a mammal without the disease.
摘要:
The present disclosure relates to liposomal particles, liposomal particles having encapsulated agents, methods of making the same, and uses thereof. Liposomal particles are useful in gene regulation and drug delivery.
摘要:
This invention demonstrated a unique cationic polymer that possesses a number features essential for systemic delivery of nucleic acids simultaneously. First, this polymer was synthesized by Zeta potential-regulated condensation of linear and branched amino group bearing oligomers through imidazole-4, 5-imine linkages, wherein the polymer diameter can be precisely adjusted from tens to hundreds nanometers by Zeta potential determinates such NaCl concentration. Most interestingly, the polymer was structured with sufficient network cavity to pack plenty copies of siRNA intramolecularly to form a polyplex of single polymer molecule. In addition, the polymer backbone degraded readily to non-toxic species and released nucleic acids in response to cellular environment due to the pKa (5.9) of its aromatically conjugated imine linage. Finally, the polyplex of single polymer molecule can be further encapsulated by a self-assembled a unilamella membrane of rationally designed triblock copolymer to which selected functional components such as cell targeting agents can be immobilized in optimized population. The lack of medicinally feasible synthetic carriers remains as the standing-hurdle blocking nucleic acids to covert from therapeutic actives to practical medicines. The present invention may serve as a "universal" vehicle to equip with variety of delivery functions for its defined structure and compatible flexibility.
摘要:
Herein described is a method for treating cancer in a subject by administering a human umbilical cord perivascular cell (HUCPVC) that has been genetically modified to increase the expression of an oligonucleotide or a polypeptide such as an anti-cancer antibody.
摘要:
The present disclosure relates to methods and compounds for promoting anabolic pathways in neuronal cells leading to improved neuronal survival. In particular, the present disclosure relates to inhibiting TSCI and or SIRT6 to promote glycolysis and neuronal survival in a variety of neurodegenerative conditions, and specifically in retinitis pigmentosa.
摘要:
Disclosed herein are three-dimensional cage molecules, wherein the cage molecule is composed of RNA. Also disclosed is a composition including the three- dimensional cage molecule, as well as a pharmaceutical composition containing the three-dimensional cage molecule. Also disclosed herein are methods of administering a cage molecule, composition, or formulation thereof to a subject in need thereof.
摘要:
Compositions including a nanocomplex of a polynucleotide with a cell-penetrating peptide and a quaternary phosphonium salt, such as tetrabutylphosphonium bromide (TBPB), are useful for delivering polynucleotides such as DNA or RNA into cells.
摘要:
The present invention relates to methods for shifting the splicing profile of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding U7-based small nuclear RNAs to induce DUX4 exon- skipping and the expression of shortened forms of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
摘要:
The present subject matter provides a method for delivering a gene editing composition across a plasma membrane of a cell. Related apparatus, systems, techniques, compositions, and articles are also described.