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9/15/2023 10:42:35 AM | Browse: 29 | Download: 11
Publication Name World Journal of Experimental Medicine
Manuscript ID 86421
Country/Territory United States
2023-06-17 19:44
Peer-Review Started
2023-06-17 19:46
To Make the First Decision
Return for Revision
2023-07-04 08:49
2023-07-16 16:47
Second Decision
2023-08-11 02:36
Accepted by Journal Editor-in-Chief
Accepted by Company Editor-in-Chief
2023-08-11 08:22
Articles in Press
2023-08-11 08:22
Publication Fee Transferred
Edit the Manuscript by Language Editor
Typeset the Manuscript
2023-09-08 15:07
Publish the Manuscript Online
2023-09-15 10:42
ISSN 2220-315x (online)
Open Access This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Copyright ©The Author(s) 2023. Published by Baishideng Publishing Group Inc. All rights reserved.
Article Reprints For details, please visit: http://www.wjgnet.com/bpg/gerinfo/247
Permissions For details, please visit: http://www.wjgnet.com/bpg/gerinfo/207
Publisher Baishideng Publishing Group Inc, 7041 Koll Center Parkway, Suite 160, Pleasanton, CA 94566, USA
Website http://www.wjgnet.com
Category Neurosciences
Manuscript Type Review
Article Title Neurologic orphan diseases: Emerging innovations and role for genetic treatments
Manuscript Source Unsolicited Manuscript
All Author List Ivelina P Kioutchoukova, Devon T Foster, Rajvi N Thakkar, Marco A Foreman, Brandon J Burgess, Rebecca M Toms, Eduardo E Molina Valero and Brandon Lucke-Wold
Author(s) ORCID Number
Ivelina P Kioutchoukova http://orcid.org/0009-0001-7467-2382
Devon T Foster http://orcid.org/0009-0007-9411-9051
Rajvi N Thakkar http://orcid.org/0009-0003-2375-5078
Marco A Foreman http://orcid.org/0000-0002-5551-7061
Brandon J Burgess http://orcid.org/0009-0008-7203-8469
Rebecca M Toms http://orcid.org/0009-0005-5539-9754
Eduardo E Molina Valero http://orcid.org/0000-0002-3235-4079
Brandon Lucke-Wold http://orcid.org/0000-0001-6577-4080
Funding Agency and Grant Number
Corresponding Author Brandon Lucke-Wold, MD, PhD, Neurosurgeon, Department of Neurosurgery, University of Florida, 1505 SW Archer Rd, Gainesville, FL 32611, United States. brandon.lucke-wold@neurosurgery.ufl.edu
Key Words Neurologic orphan diseases; gene therapy; Clustered regularly interspaced palindromic repeats/Cas9; Antisense oligonucleotides; Adeno-associated virus; mTOR inhibitors
Core Tip Neurologic orphan diseases are rare conditions that impact a small percentage of the population. Through new advances in technology and research, the use of genetic treatment for these conditions is increasing. Recent advances in clustered regularly interspaced palindromic repeats/Cas9, adeno-associated viral vectors, antisense oligonucleotides, and mammalian target of rapamycin inhibitors have shown improvements in the care of patients and their quality of life.
Publish Date 2023-09-15 10:42
Citation Kioutchoukova IP, Foster DT, Thakkar RN, Foreman MA, Burgess BJ, Toms RM, Molina Valero EE, Lucke-Wold B. Neurologic orphan diseases: Emerging innovations and role for genetic treatments. World J Exp Med 2023; 13(4): 59-74
URL https://www.wjgnet.com/2220-315x/full/v13/i4/59.htm
DOI https://dx.doi.org/10.5493/wjem.v13.i4.59
Full Article (PDF) WJEM-13-59-with-cover.pdf
Full Article (Word) WJEM-13-59.docx
Manuscript File 86421_Auto_Edited-LJH-JLW.docx
Answering Reviewers 86421-Answering reviewers.pdf
Audio Core Tip 86421-Audio core tip.mp3
Conflict-of-Interest Disclosure Form 86421-Conflict-of-interest statement.pdf
Copyright License Agreement 86421-Copyright license agreement.pdf
Peer-review Report 86421-Peer-review(s).pdf
Scientific Misconduct Check 86421-Bing-Liu JH-2.png
Scientific Misconduct Check 86421-CrossCheck.png
Scientific Editor Work List 86421-Scientific editor work list.pdf