Delivering the Promise of Gene Therapy with Nanomedicines in Treating Central Nervous System Diseases

Abstract

Central Nervous System (CNS) diseases, such as Alzheimer's diseases (AD), Parkinson's Diseases (PD), brain tumors, Huntington's disease (HD), and stroke, still remain difficult to treat by the conventional molecular drugs. In recent years, various gene therapies have come into the spotlight as versatile therapeutics providing the potential to prevent and treat these diseases. Despite the significant progress that has undoubtedly been achieved in terms of the design and modification of genetic modulators with desired potency and minimized unwanted immune responses, the efficient and safe in vivo delivery of gene therapies still poses major translational challenges. Various non‐viral nanomedicines have been recently explored to circumvent this limitation. In this review, an overview of gene therapies for CNS diseases is provided and describes recent advances in the development of nanomedicines, including their unique characteristics, chemical modifications, bioconjugations, and the specific applications that those nanomedicines are harnessed to deliver gene therapies.

Nucleic acid delivery by ionizable nanocarriers for brain disease treatment

“…The advent of gene therapy and the rapid advancement of genome engineering technology have opened up new avenues for the treatment of CNS diseases through the use of INs to encapsulate nucleic acids, such as siRNA, miRNA, and pDNA, or other synthetic drugs. This approach has the potential to effectively target brain tissue and treat CNS diseases [1] …”

“…Brain diseases are a major global health burden including a wide range of conditions, such as acute disseminated encephalomyelitis (infections), cerebral atrophy (trauma), epilepsy (seizures), gliomas (tumors and masses), stroke (vascular conditions), multiple sclerosis (autoimmune conditions), and Alzheimer's disease (AD), neurodegenerative conditions. [1] Despite recent advances in drug development, brain disease therapies still have a low success rate due to less understanding of the major barrier of the brain tissue, which make it challenging for molecules to reach the brain.…”

“…Brain diseases are a major global health burden including a wide range of conditions, such as acute disseminated encephalomyelitis (infections), cerebral atrophy (trauma), epilepsy (seizures), gliomas (tumors and masses), stroke (vascular conditions), multiple sclerosis (autoimmune conditions), and Alzheimer's disease (AD), neurodegenerative conditions [1] …”

A Multifunctional Porous Silicon Nanocarrier for Glioblastoma Treatment

“…Porous silicon nanoparticles (pSiNPs) provide salient advantages as nanocarriers as they have high cargo loading capacity, tunable size and pore size, and good biocompatibility . They naturally and completely degrade to nontoxic orthosilicic acid.…”

“…Porous silicon nanoparticles (pSiNPs) provide salient advantages as nanocarriers as they have high cargo loading capacity, tunable size and pore size, and good biocompatibility. 12 They naturally and completely degrade to nontoxic orthosilicic acid. Additionally, pSiNPs have versatile surface allowing for various modifications, such as targeting ligands, insulin, dendrimers, antibodies and polymers.…”