Ansh Patel

Review of Nanoparticle-Based Drug Delivery Methods for the Treatment of Spinal Muscular Atrophy

Started May 18, 2022

Abstract or project description

Nanoparticles (NP) have shown great potential as drug delivery systems that are highly effective while also enhancing the chemical properties of the drug itself. In the last decade, numerous studies and clinical trials have shown that nanotechnology-based strategies are significantly efficient and versatile in the field of biomedicine especially as nanocarriers for treatments in rare neural, cardiovascular and blood disorders. Nanoparticle-based drug delivery systems are especially prominent solutions for neurological diseases because of the blood-brain barrier (BBB) and blood-cerebrospinal fluid barrier (BCSFB) that both block most endogenous and exogenous substances from entering into the central nervous system (CNS). Spinal Muscular Atrophy (SMA) is a rare genetic neurodegenerative disease affecting lower motor neurons and is the leading cause of genetic infant mortality. Antisense Oligonucleotides (ASOs) are a genetic treatment for SMA by split-slicing pre-messenger RNA (pre-mRNA) and halting protein translation. Due to its poor penetration and low cellular uptake, it is inefficacious when trying to mediate sites in the CNS. However, cell-penetrating peptide nanoparticles (CPPs) have shown to be significant nanocarriers that enhance the drug’s chemical properties in offsetting the disease and allow it to reach into the CNS with increased target specification. This review provides a discussion of advancement in nanotechnology-based drug delivery systems specified to amelioration of ASOs relevant to SMA disease progression and its usage in developing further treatment for SMA patients.