TY  - GEN
AV  - public
N2  - Leber congenital amaurosis (LCA) caused by AIPL1 mutations is one of the most severe forms of inherited retinal degeneration (IRD). The rapid and extensive photoreceptor degeneration challenges the development of potential treatments. Nevertheless, preclinical studies show that both gene augmentation and photoreceptor transplantation can regenerate and restore retinal function in animal models of AIPL1-associated LCA. However, questions regarding long-term benefit and safety still remain as these therapies advance towards clinical application. Ground-breaking advances in stem cell technology and genome editing are examples of alternative therapeutic approaches and address some of the limitations associated with previous methods. The continuous development of these cutting-edge biotechnologies paves the way towards a bright future not only for AIPL1-associated LCA patients but also other forms of IRD.
KW  - Adeno-associated virus (AAV)
KW  -  Aryl hydrocarbon receptor-interacting protein-like 1 (AIPL1)
KW  -  CRISPR/Cas9
KW  -  Cell therapy
KW  -  Gene therapy
KW  -  Genome editing
KW  -  Leber congenital amaurosis (LCA)
KW  -  Photoreceptor transplantation
KW  -  Retinal degeneration
KW  -  Stem cell
A1  - Perdigao, PRL
A1  - van der Spuy, J
EP  - 101
SP  - 97
T3  - Advances in Experimental Medicine and Biology
PB  - Springer
UR  - https://doi.org/10.1007/978-3-030-27378-1_16
Y1  - 2019/12/29/
ID  - discovery10090079
CY  - Cham, Switzerland
N1  - This version is the author accepted manuscript. For information on re-use, please refer to the publisher?s terms and conditions.
TI  - Gene and Cell Therapy for AIPL1-Associated Leber Congenital Amaurosis: Challenges and Prospects
ER  -