PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF DYNAMIN-1 VARIANTS

WIPO Patent Application WO/2022/221574

A3

RNA interference-based methods and products for inhibiting the expression of pathogenic dynamin-1 variants are provided. Delivery vehicles such as recombinant adeno- associated viruses deliver DNAs encoding RNAs that inhibit the expression of the dynamin-1 variants. The methods treat, for example, developmental and epileptic encephalopathies.

WO/2024/048528	PROPHYLACTIC AGENT AND/OR THERAPEUTIC AGENT FOR HEART DISEASES AND LAMINOPATHY
JP4456169	Signals for packaging influenza viral vectors
JP2024507394	Formulations for aerosol formation and aerosols for delivering nucleic acids

HARPER SCOTT (US)
FRANKEL WAYNE (US)

PCT/US2022/024885

February 23, 2023

April 14, 2022

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RES INST NATIONWIDE CHILDRENS HOSPITAL (US)
UNIV COLUMBIA (US)

A61K31/7105; A61K31/7088; A61P25/28; C12N15/113

US20070197438A1

2007-08-23

AIMIUWU OSASUMWEN V., FOWLER ALLISON M., SAH MEGHA, TEOH JIA JIE, KANBER AYLA, PYNE NETTIE K., PETRI SABRINA, ROSENTHAL-WEISS CHAN: "RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model", MOLECULAR THERAPY, ELSEVIER INC., US, vol. 28, no. 7, 1 July 2020 (2020-07-01), US , pages 1706 - 1716, XP093037753, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2020.04.007
NIDETZ NATALIE F.; MCGEE MICHAEL C.; TSE LONGPING V.; LI CHENGWEN; CONG LE; LI YUNXING; HUANG WEISHAN: "Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery", PHARMACOLOGY & THERAPEUTICS, ELSEVIER., GB, vol. 207, 11 December 2019 (2019-12-11), GB , XP086060316, ISSN: 0163-7258, DOI: 10.1016/j.pharmthera.2019.107453

NOLAND, Greta, E. (US)

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