TY - JOUR
T1 - siRNA therapeutics and its challenges
T2 - Recent advances in effective delivery for cancer therapy
AU - Paul, Arpita
AU - Muralidharan, Anuraag
AU - Biswas, Avirup
AU - Kamath, B. Venkatesh
AU - Joseph, Alex
AU - Alex, Angel Treasa
N1 - Funding Information:
The authors are grateful and would like to acknowledge Indian Council of Medical Research (ICMR), Government of India, New Delhi for Senior Research Fellowship to Ms. Arpita Paul. The authors are grateful to ICMR, Government of India, New Delhi, and Manipal College of Pharmaceutical Sciences, Manipal Academy of Higher Education, Manipal for providing financial and infrastructural support for the research activities.
Publisher Copyright:
© 2022
PY - 2022/7/1
Y1 - 2022/7/1
N2 - Small interfering RNA (siRNA) has emerged as a promising strategy for treating several “undruggable” genetically impaired conditions. The siRNA-based drugs have opened the doors to a novel mode of therapy. Though siRNA is a potential therapeutic strategy to treat various disorders, its application is still not widely accepted due to certain drawbacks such as off-target effects, stability etc. To overcome these hurdles, several promising modifications and strategies have been reported. Cancer has been identified to have several involvement of genetic imbalances, where siRNA-based formulations have shown promising results both in vitro and in vivo. Apart from cancer, orphan diseases too have an urgent need for novel and effective remedies which is slowly being fulfilled by the chemically synthesized siRNA therapeutics. Till date five siRNA-based drugs namely Patisiran, Givlaari, Oxlumo™, Leqvio and Amvuttra have been approved by USFDA, which have initiated a new category of biopharmaceuticals in the market.
AB - Small interfering RNA (siRNA) has emerged as a promising strategy for treating several “undruggable” genetically impaired conditions. The siRNA-based drugs have opened the doors to a novel mode of therapy. Though siRNA is a potential therapeutic strategy to treat various disorders, its application is still not widely accepted due to certain drawbacks such as off-target effects, stability etc. To overcome these hurdles, several promising modifications and strategies have been reported. Cancer has been identified to have several involvement of genetic imbalances, where siRNA-based formulations have shown promising results both in vitro and in vivo. Apart from cancer, orphan diseases too have an urgent need for novel and effective remedies which is slowly being fulfilled by the chemically synthesized siRNA therapeutics. Till date five siRNA-based drugs namely Patisiran, Givlaari, Oxlumo™, Leqvio and Amvuttra have been approved by USFDA, which have initiated a new category of biopharmaceuticals in the market.
UR - http://www.scopus.com/inward/record.url?scp=85135940276&partnerID=8YFLogxK
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U2 - 10.1016/j.onano.2022.100063
DO - 10.1016/j.onano.2022.100063
M3 - Review article
AN - SCOPUS:85135940276
SN - 2352-9520
VL - 7
JO - OpenNano
JF - OpenNano
M1 - 100063
ER -
