ADMINISTRACION DE ACIDO NUCLEICO AL SISTEMA NERVIOSO CENTRAL

• Main Authors: Vishwesh A. PATIL, PENG Paul, BORNAPOUR Mandana, Brian R. SIMLER, Hasan M. YILDIZ, William F. KIESMAN
• Format: Patent
• Language: Spanish
• Published: 31.10.2022
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

Se presentan nanoportadores poliméricos (p. ej., nanopartículas de PLGA) con ácido nucleico encapsulado (p. ej., un oligonucleótido antisentido) para la administración (p. ej., por vía intratecal) al sistema nervioso central. Estos nanoportadores poliméricos son útiles en el tratamiento de trastornos del sistema nervioso central. Son capaces de administrar su carga (p. ej., un oligonucleótido antisentido) en cantidades mayores, durante un período de tiempo más largo y en regiones más profundas del cerebro que un oligonucleótido antisentido libre o no formulado. La administración y distribución eficientes del oligonucleótido antisentido resulta en la reducción del número de administraciones. Featured are polymeric nanocarriers (e.g., PLGA nanoparticles) with encapsulated nucleic acid (e.g., an antisense oligonucleotide) for delivery (e.g., intrathecally) to the central nervous system. These polymeric nanocarriers are useful in the treatment of central nervous system disorders. They are capable of delivering their cargo (e.g., an antisense oligonucleotide) in higher amounts, for a longer period of time, and into deeper regions of the brain than a free or unformulated antisense oligonucleotide. The efficient delivery and distribution of antisense oligonucleotides results in reducing the number of administrations and patient compliance and improves patient experience.