Review
Copyright ©The Author(s) 2023. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Exp Med. Sep 20, 2023; 13(4): 59-74
Published online Sep 20, 2023. doi: 10.5493/wjem.v13.i4.59
Neurologic orphan diseases: Emerging innovations and role for genetic treatments
Ivelina P Kioutchoukova, Devon T Foster, Rajvi N Thakkar, Marco A Foreman, Brandon J Burgess, Rebecca M Toms, Eduardo E Molina Valero, Brandon Lucke-Wold
Ivelina P Kioutchoukova, Rajvi N Thakkar, Marco A Foreman, Brandon J Burgess, Rebecca M Toms, Eduardo E Molina Valero, College of Medicine, University of Florida, Gainesville, FL 32611, United States
Devon T Foster, Florida International University Herbert Wertheim College of Medicine, Florida International University Herbert Wertheim College of Medicine, Miami, FL 33199, United States
Brandon Lucke-Wold, Department of Neurosurgery, University of Florida, Gainesville, FL 32611, United States
Author contributions: Lucke-Wold B contributed to conceptualization and supervision; Kioutchoukova I, Foster D, Thakkar R, Foreman M, Burgess B, Toms R, Valero EM contributed to writing-original draft presentation; Ivelina Kioutchoukova, Devon Foster contributed to writing-editing and review.
Conflict-of-interest statement: All the authors declare no conflict of interest.
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: https://creativecommons.org/Licenses/by-nc/4.0/
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
Received: June 17, 2023
Peer-review started: June 17, 2023
First decision: July 4, 2023
Revised: July 16, 2023
Accepted: August 11, 2023
Article in press: August 11, 2023
Published online: September 20, 2023
Processing time: 89 Days and 15 Hours
Core Tip

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.