JOM KITA KE POLITEKNIK

Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy (Record no. 1954)

MARC details
042 ## - AUTHENTICATION CODE
Authentication code dc
100 10 - MAIN ENTRY--PERSONAL NAME
Personal name Hammond, Suzan M.
Relator term author
9 (RLIN) 2385
245 00 - TITLE STATEMENT
Title Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy
260 ## - PUBLICATION, DISTRIBUTION, ETC.
Name of publisher, distributor, etc. American Society for Clinical Investigation,
Date of publication, distribution, etc. 2022-12-22.
500 ## - GENERAL NOTE
General note /pmc/articles/PMC7614086/
500 ## - GENERAL NOTE
General note /pubmed/36346674
520 ## - SUMMARY, ETC.
Summary, etc. Antisense oligonucleotides (ASOs) have emerged as one of the most innovative new genetic drug modalities. However, their high molecular weight limits their bioavailability for otherwise-treatable neurological disorders. We investigated conjugation of ASOs to an antibody against the murine transferrin receptor, 8D3(130), and evaluated it via systemic administration in mouse models of the neurodegenerative disease spinal muscular atrophy (SMA). SMA, like several other neurological and neuromuscular diseases, is treatable with single-stranded ASOs that modulate splicing of the survival motor neuron 2 (SMN2) gene. Administration of 8D3(130)-ASO conjugate resulted in elevated levels of bioavailability to the brain. Additionally, 8D3(130)-ASO yielded therapeutic levels of SMN2 splicing in the central nervous system of adult human SMN2-transgenic (hSMN2-transgenic) mice, which resulted in extended survival of a severely affected SMA mouse model. Systemic delivery of nucleic acid therapies with brain-targeting antibodies offers powerful translational potential for future treatments of neuromuscular and neurodegenerative diseases.
540 ## - TERMS GOVERNING USE AND REPRODUCTION NOTE
Terms governing use and reproduction © 2022 Hammond et al.
540 ## - TERMS GOVERNING USE AND REPRODUCTION NOTE
Terms governing use and reproduction https://creativecommons.org/licenses/by/4.0/This work is licensed under the Creative Commons Attribution 4.0 International License. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) .
546 ## - LANGUAGE NOTE
Language note en
690 ## - LOCAL SUBJECT ADDED ENTRY--TOPICAL TERM (OCLC, RLIN)
Topical term or geographic name as entry element Research Article
9 (RLIN) 1689
655 7# - INDEX TERM--GENRE/FORM
Genre/form data or focus term Text
Source of term local
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Abendroth, Frank
Relator term author
9 (RLIN) 2386
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Goli, Larissa
Relator term author
9 (RLIN) 2387
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Stoodley, Jessica
Relator term author
9 (RLIN) 2388
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Burrell, Matthew
Relator term author
9 (RLIN) 2389
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Thom, George
Relator term author
9 (RLIN) 2390
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Gurrell, Ian
Relator term author
9 (RLIN) 2391
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Ahlskog, Nina
Relator term author
9 (RLIN) 2392
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Gait, Michael J.
Relator term author
9 (RLIN) 2393
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Wood, Matthew J.A.
Relator term author
9 (RLIN) 2394
700 10 - ADDED ENTRY--PERSONAL NAME
Personal name Webster, Carl I.
Relator term author
9 (RLIN) 2395
786 0# - DATA SOURCE ENTRY
Note JCI Insight
856 41 - ELECTRONIC LOCATION AND ACCESS
Uniform Resource Identifier <a href="http://dx.doi.org/10.1172/jci.insight.154142">http://dx.doi.org/10.1172/jci.insight.154142</a>
Public note Connect to this object online.

No items available.