Results 241 to 250 of about 266,614 (380)

Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing

Small, EarlyView.
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 more
wiley   +1 more source

An antisense oligonucleotide-based strategy to ameliorate cognitive dysfunction in the 22q11.2 Deletion Syndrome. [PDF]

Elife
Thakur P, Lackinger M, Diamantopoulou A, Rao S, Chen Y, Khalizova K, Ferng A, Mazur C, Kordasiewicz H, Shprintzen RJ, Markx S, Xu B, Gogos JA.   +12 more
europepmc   +1 more source

Targeted Delivery of miR‐34a via Anti‐CD47 Antibody Conjugates for Enhanced Cancer Immunotherapy in Triple Negative Breast Cancer

Small, EarlyView.
aCD47‐C‐miR34a, an antibody‐oligonucleotide platform combining an anti‐CD47 antibody with miR‐34a, is designed for targeted therapy in TNBC. This approach improved delivery and restored the tumor‐suppressing function of miR‐34a, enhanced CD8+ T‐cell activation, and inhibited tumor growth, providing a potential strategy to overcome immune evasion and ...
Youngri Ryu, Eun Hye Kim, Hochung Jang, Yelee Kim, Byeongmin Park, Jiwoong Choi, Yeongji Jang, Sung‐Gil Chi, Man Kyu Shim, Sun Hwa Kim, Hong Yeol Yoon, Yoosoo Yang   +11 more
wiley   +1 more source

In vivo electroporetic transfer ofbcl-2 antisense oligonucleotide inhibits the development of hepatocellular carcinoma in rats [PDF]

, 2000
Miyako Baba, Hiroyasu Iishi, Masaharu Tatsuta   +2 more
openalex   +2 more sources

Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment. [PDF]

Beilstein J Nanotechnol
Alvarez AC, Maguire D, Brannigan RP.
europepmc   +1 more source

Fluorescence imaging of MALAT1 expression using a Cy5.5-labeled antisense oligonucleotide in lung cancer and epidermal carcinoma cells. [PDF]

Cancer Imaging
Liu Z, Yao C, Ni H, Wang G, Dong M.
europepmc   +1 more source

Targeting KRAS-dependent tumors with AZD4785, a high-affinity therapeutic antisense oligonucleotide inhibitor of KRAS

Science Translational Medicine, 2017
Sarah J. Ross, A. Revenko, Lyndsey Hanson, Rebecca Ellston, A. Staniszewska, Nicky Whalley, Sanjay K. Pandey, M. Revill, C. Rooney, Linda K. Buckett, Stephanie K. Klein, K. Hudson, Brett P. Monia, M. Zinda, David C. Blakey, Paul D. Lyne, A. Macleod   +16 more
semanticscholar   +1 more source

Patient‐Reported Outcomes in the Phase III OASIS‐HAE Study of Donidalorsen for Hereditary Angioedema

Allergy, EarlyView.
We report an overview of patients' self‐reported quality of life, disease control, and functioning in daily life while taking donidalorsen for HAE in the 24‐week phase 3 OASIS‐HAE trial. Most patients reported clinically meaningful improvements in quality of life and disease control.
Marc A. Riedl, Aaron Yarlas, Laura Bordone, Sabrina Treadwell, Sophie Wang, Kenneth B. Newman, Danny M. Cohn   +6 more
wiley   +1 more source

Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments. [PDF]

Am J Hum Genet
Cheerie D, Meserve MM, Beijer D, Kaiwar C, Newton L, Taylor Tavares AL, Verran AS, Sherrill E, Leonard S, Sanders SJ, Blake E, Elkhateeb N, Gandhi A, Liang NSY, Morgan JT, Verwillow A, Verheijen J, Giles A, Williams S, Chopra M, Croft L, Dafsari HS, Davidson AE, Friedman J, Gregor A, Haque B, Lechner R, Montgomery KA, Ryten M, Schober E, Siegel G, Sullivan PJ, Whittle EF, Zardetto B, Yu TW, Synofzik M, Aartsma-Rus A, Costain G, Lauffer MC, N=1 Collaborative.   +39 more
europepmc   +1 more source

Discovery of natural product derivative triptolidiol as a direct NLRP3 inhibitor by reducing K63‐specific ubiquitination

British Journal of Pharmacology, EarlyView.
▪ Background and Purpose NLRP3 is up‐regulated in inflammatory and autoimmune diseases. The development of NLRP3 inhibitors is challenged by the identification of compounds with distinct mechanisms of action avoiding side effects and toxicity. Triptolide is a natural product with multiple anti‐inflammatory activities, but a narrow therapeutic window ...
Mo‐Yu Ding, Chengqing Ning, Shao‐Ru Chen, Hao‐Ran Yin, Jing Xu, Ying Wang   +5 more
wiley   +1 more source