Warning: imagejpeg(C:\Inetpub\vhosts\kidney.de\httpdocs\phplern\27376675
.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 Exp+Dermatol
2017 ; 26
(1
): 3-10
Nephropedia Template TP
gab.com Text
Twit Text FOAVip
Twit Text #
English Wikipedia
RNA-based therapies for genodermatoses
#MMPMID27376675
Bornert O
; Peking P
; Bremer J
; Koller U
; van den Akker PC
; Aartsma-Rus A
; Pasmooij AM
; Murauer EM
; Nyström A
Exp Dermatol
2017[Jan]; 26
(1
): 3-10
PMID27376675
show ga
Genetic disorders affecting the skin, genodermatoses, constitute a large and
heterogeneous group of diseases, for which treatment is generally limited to
management of symptoms. RNA-based therapies are emerging as a powerful tool to
treat genodermatoses. In this review, we discuss in detail RNA splicing
modulation by antisense oligonucleotides and RNA trans-splicing, transcript
replacement and genome editing by in vitro-transcribed mRNAs, and gene knockdown
by small interfering RNA and antisense oligonucleotides. We present the current
state of these therapeutic approaches and critically discuss their opportunities,
limitations and the challenges that remain to be solved. The aim of this review
was to set the stage for the development of new and better therapies to improve
the lives of patients and families affected by a genodermatosis.
|Animals
[MESH]
|Gene Knockdown Techniques
[MESH]
|Genetic Therapy/*methods
[MESH]
|Humans
[MESH]
|Oligonucleotides, Antisense/therapeutic use
[MESH]
free
free
free
