Deprecated: Implicit conversion from float 217.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534
Deprecated: Implicit conversion from float 217.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534
Warning: imagejpeg(C:\Inetpub\vhosts\kidney.de\httpdocs\phplern\29766738
.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 Cell+Transplant
2018 ; 27
(3
): 364-378
Nephropedia Template TP
gab.com Text
Twit Text FOAVip
Twit Text #
English Wikipedia
The Role of Gene Editing in Neurodegenerative Diseases
#MMPMID29766738
Fan HC
; Chi CS
; Lee YJ
; Tsai JD
; Lin SZ
; Harn HJ
Cell Transplant
2018[Mar]; 27
(3
): 364-378
PMID29766738
show ga
Neurodegenerative diseases (NDs), at least including Alzheimer's, Huntington's,
and Parkinson's diseases, have become the most dreaded maladies because there are
no precise diagnostic tools or definite treatments for these debilitating
diseases. The increased prevalence and a substantial impact on the
social-economic and medical care of NDs propel governments to develop policies to
counteract the impact. Although the etiologies of NDs are still unknown, growing
evidence suggests that genetic, cellular, and circuit alternations may cause the
generation of abnormal misfolded proteins, which uncontrolledly accumulate to
damage and eventually overwhelm the protein-disposal mechanisms of these neurons,
leading to a common pathological feature of NDs. If the functions and the
connectivity can be restored, alterations and accumulated damages may improve.
The gene-editing tools including zinc-finger nucleases (ZFNs), transcription
activator-like effector nucleases (TALENs), and clustered regularly interspaced
short palindromic repeats-associated nucleases (CRISPR/CAS) have emerged as a
novel tool not only for generating specific ND animal models for interrogating
the mechanisms and screening potential drugs against NDs but also for the editing
sequence-specific genes to help patients with NDs to regain function and
connectivity. This review introduces the clinical manifestations of three
distinct NDs and the applications of the gene-editing technology on these
debilitating diseases.
|*Gene Editing
[MESH]
|Clustered Regularly Interspaced Short Palindromic Repeats/genetics
[MESH]