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Splice-switching antisense oligonucleotide-mediated knockdown of ATXN2 for the potential treatment of amyotrophic lateral sclerosis
Thesis   Open access

Splice-switching antisense oligonucleotide-mediated knockdown of ATXN2 for the potential treatment of amyotrophic lateral sclerosis

Alanis Lima
Honours, Murdoch University
2022
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Open Access

Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive degenerative disease of motor neurons in the central nervous system, that often manifests from the age of 40 to 70 years and results in paralysis and death within 3 to 5 years of symptom onset (1). ALS is estimated to affect between 1.57 and 9.62 per 100,000 individuals (2). The cost of ALS in 2015 was estimated to be $2.37 billion Australia-wide, presenting a significant economic burden (3). Furthermore, the global incidence of ALS is projected to increase by 69% from 2015 to 2040, as the proportion of individuals aged over 64 is expected to rise to 16% by 2050, from 9% in 2015 (4, 5)...

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