scholarly journals Antisense Oligonucleotides: Pharmacology and Delivery Strategies

2020 ◽  
Vol 5 (01) ◽  
pp. 7-11
Author(s):  
Mohammed Abdul Samad ◽  
Kavya Pandiri ◽  
Anjani Prasad Bojanapally
Keyword(s):  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotides ◽  
Delivery Methods ◽  
Fundamental Idea ◽  
Therapeutic Outcomes ◽  
Wide Range ◽  
Effective Delivery ◽  
Delivery Strategies ◽  
Antisense Oligonucleotide Therapy ◽  
Modify Protein

Antisense oligonucleotide therapy is a dominant drug discovery approach that can explicitly modify protein synthesis through numerous mechanisms. The limitations of antisense oligonucleotide (ASO) therapy in delivery strategies have been overcome in recent years with different ligands carriers, as well as, through nanocarriers. ASO therapy was successfully applied towards targeting a wide range of therapeutic areas. There is an expanding enthusiasm in extending the utilization of antisense compounds to numerous different diseases due to their safe and potential therapeutic outcomes. Thus, the present review attempted to elaborate on the fundamental idea of antisense technology, approaches, and safe and effective delivery methods.

scholarly journals Medicinal Applications of Antisense Oligonucleotides: A Review

2020 ◽  
Vol 5 (02) ◽  
pp. 30-36
Author(s):  
Kavya Pandiri ◽  
Mohammed Abdul Samad ◽  
Nadeem Abbas Gulamus ◽  
Hajera Khanam
Keyword(s):  
High Throughput ◽  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotides ◽  
Therapeutic Approach ◽  
Antisense Technology ◽  
Dna And Rna ◽  
Wide Range ◽  
High Throughput Method ◽  
Delivery Strategies ◽  
Antisense Oligonucleotide Therapy

Antisense technology has emerged as a fast and conceivably high-throughput method for repressing genes due to advancement in knowledge about DNA and RNA physiology. The limitations of antisense oligonucleotide therapy in delivery strategies have been overcome in recent years. Antisense oligonucleotide treatment was effectively applied towards targeting a wide range of therapeutic areas. With ongoing approvals of antisense oligonucleotides, there is an expanding enthusiasm for increasing the utilization of these compounds for curing various infections. This short survey gives a far-reaching comprehension of applications of antisense technology, how they can be utilized therapeutically, and current endeavors to grow new antisense oligonucleotide treatments that will add a forthcoming therapeutic approach for the treatment of various diseases.

scholarly journals Wrangling RNA: Antisense oligonucleotides for neurological disorders

2019 ◽  
Vol 11 (511) ◽  
pp. eaay2069 ◽  
Author(s):  
Kevin Talbot ◽  
Matthew J. A. Wood
Keyword(s):  
Spinal Muscular Atrophy ◽  
Effective Treatment ◽  
Neurological Disorders ◽  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotides ◽  
Muscular Atrophy ◽  
Antisense Oligonucleotide Therapy

Effective treatment of spinal muscular atrophy with antisense oligonucleotide therapy opens the door to treating other neurological disorders with this approach.

scholarly journals Cationic liposome-encapsulated antisense oligonucleotide mediates efficient killing of intracellular Leishmania

1999 ◽  
Vol 340 (2) ◽  
pp. 393-396 ◽  
Author(s):  
Rila CHAKRABORTY ◽  
Dalia DASGUPTA ◽  
Samit ADHYA ◽  
Mukul K. BASU
Keyword(s):  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotides ◽  
Cationic Liposome ◽  
Cationic Liposomes ◽  
Antiparasitic Activity ◽  
Intracellular Parasites ◽  
Low Concentrations ◽  
Effective Delivery

Antisense oligonucleotides have been considered as inhibitors of growth of intracellular parasites such as Leishmania, but only limited inhibition has been observed in vitro. We have encapsulated an antisense oligonucleotide, complementary to the Leishmania universal miniexon sequence, in cationic liposomes. Low concentrations (4 μM) of encapsulated oligonucleotides specifically reduced the amastigote burden within cultured macrophages by 80%. This result illustrates the importance of effective delivery for efficient antiparasitic activity of antisense oligonucleotides.

Dysregulation of microRNAs in spinal muscular atrophy and the response to antisense oligonucleotide therapy

2015 ◽  
Vol 25 ◽  
pp. S194
Author(s):  
H. Zhou ◽  
F. Catapano ◽  
I. Zaharieva ◽  
M. Scoto ◽  
J. Morgan ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Antisense Oligonucleotide ◽  
Muscular Atrophy ◽  
Antisense Oligonucleotide Therapy

Antisense-Oligonucleotide Therapy

1996 ◽  
Vol 334 (5) ◽  
pp. 316-318 ◽  
Author(s):  
Frederick K. Askari ◽  
W. Michael McDonnell
Keyword(s):  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotide Therapy

scholarly journals LOX-1 Inhibition in ApoE KO Mice Using a Schizophyllan-based Antisense Oligonucleotide Therapy

2012 ◽  
Vol 1 ◽  
pp. e58 ◽  
Author(s):  
Francesca Amati ◽  
Laura Diano ◽  
Lucia Vecchione ◽  
Giuseppe Danilo Norata ◽  
Yoshikazu Koyama ◽  
...  
Keyword(s):  
Antisense Oligonucleotide ◽  
Apoe Ko Mice ◽  
Antisense Oligonucleotide Therapy ◽  
Apoe Ko

RNAi-Mediated Screening of Selected Target Genes Against Culex quinquefasciatus (Diptera: Culicidae)

2021 ◽  
Author(s):  
Sayed M S Khalil ◽  
Kashif Munawar ◽  
Azzam M Alahmed ◽  
Ahmed M A Mohammed
Keyword(s):  
Culex Quinquefasciatus ◽  
Target Genes ◽  
Mosquito Control ◽  
Larval Mortality ◽  
Adult Emergence ◽  
Lethal Effect ◽  
Delivery Methods ◽  
Valley Fever ◽  
Wide Range ◽  
Lethal Genes

Abstract Culex quinquefasciatus, a member of the Culex pipiens complex, is widespread in Saudi Arabia and other parts of the world. It is a vector for lymphatic filariasis, Rift Valley fever, and West Nile virus. Studies have shown the deleterious effect of RNA interference (RNAi)-mediated knockdown of various lethal genes in model and agricultural pest insects. RNAi was proposed as a tool for mosquito control with a focus on Aedes aegypti and Anopheles gambiae. In this study, we examined the effect of RNAi of selected target genes on both larval mortality and adult emergence of Cx. quinquefasciatus through two delivery methods: soaking and nanoparticles. Ten candidate genes were selected for RNAi based on their known lethal effect in other insects. Disruption of three genes, chitin synthase-1, inhibitor of apoptosis 1, and vacuolar adenosine triphosphatase, resulted in the highest mortality among the selected genes using the two treatment methods. Silencing the other seven genes resulted in a medium to low mortality in both assays. These three genes are also active against a wide range of insects and could be used for RNAi-based mosquito control in the future.

Congenital disorders

2018 ◽  
pp. 160-163
Author(s):  
Sowmiya Moorthie
Keyword(s):  
Developmental Disorders ◽  
Genetic Disorders ◽  
Holistic Approach ◽  
Congenital Disorders ◽  
Mortality And Morbidity ◽  
Specialist Services ◽  
Wide Range ◽  
Effective Delivery ◽  
The Life Course ◽  
Preventative Services

Congenital disorders encompass a wide range of conditions (e.g. genetic disorders, foetal disease, and developmental disorders) that occur before birth and are an important contributor to mortality and morbidity worldwide. Congenital disorders can be identified at different life stages and effective health services take a holistic approach to their care and prevention. This involves both population health and specialist services across the life course. Systematic collection of data on the types, prevalence, severity, and outcomes of congenital disorders, along with analysis and interpretation of data helps to inform appropriate planning of care and preventative services and activities. Important concepts in relation to congenital disorders, prevention activities, and key challenges to their effective delivery are described in this chapter.

scholarly journals Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

2019 ◽  
Vol 42 (1) ◽  
pp. 385-406 ◽  
Author(s):  
C. Frank Bennett ◽  
Adrian R. Krainer ◽  
Don W. Cleveland
Keyword(s):  
Neurodegenerative Diseases ◽  
Spinal Muscular Atrophy ◽  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotides ◽  
Muscular Atrophy ◽  
Neurological Diseases ◽  
Great Promise ◽  
Disease Symptoms ◽  
Therapeutic Platform ◽  
Lateral Sclerosis

Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases. Antisense drugs are currently in development for the treatment of amyotrophic lateral sclerosis, Huntington's disease, and Alzheimer's disease, and multiple research programs are underway for additional neurodegenerative diseases. One antisense drug, nusinersen, has been approved for the treatment of spinal muscular atrophy. Importantly, nusinersen improves disease symptoms when administered to symptomatic patients rather than just slowing the progression of the disease. In addition to the benefit to spinal muscular atrophy patients, there are discoveries from nusinersen that can be applied to other neurological diseases, including method of delivery, doses, tolerability of intrathecally delivered antisense drugs, and the biodistribution of intrathecal dosed antisense drugs. Based in part on the early success of nusinersen, antisense drugs hold great promise as a therapeutic platform for the treatment of neurological diseases.

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