CRISPR: a tool with potential for genomic reprogramming in neurological disorders

Dhuriya, Yogesh K.; Naik, Aijaz Ahmad

doi:10.1007/s11033-022-08136-z

Cited by 3 publications

(2 citation statements)

References 100 publications

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“…By targeting genes associated with ion channels, neurotransmitter receptors, or cellular signaling pathways, researchers can investigate how alterations in these genes contribute to epileptogenesis [ 195 , 196 ]. Additionally, gene editing tools are being used to correct disease-causing mutations in patient-derived cells or animal models, potentially paving the way for personalized gene therapies tailored to specific genetic defects [ 197 , 198 , 199 , 200 ].…”

Section: Gene Therapies For Epilepsymentioning

confidence: 99%

A Comprehensive Review of Emerging Trends and Innovative Therapies in Epilepsy Management

Ghosh,

Sinha,

Ghosh

et al. 2023

Brain Sciences

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Epilepsy is a complex neurological disorder affecting millions worldwide, with a substantial number of patients facing drug-resistant epilepsy. This comprehensive review explores innovative therapies for epilepsy management, focusing on their principles, clinical evidence, and potential applications. Traditional antiseizure medications (ASMs) form the cornerstone of epilepsy treatment, but their limitations necessitate alternative approaches. The review delves into cutting-edge therapies such as responsive neurostimulation (RNS), vagus nerve stimulation (VNS), and deep brain stimulation (DBS), highlighting their mechanisms of action and promising clinical outcomes. Additionally, the potential of gene therapies and optogenetics in epilepsy research is discussed, revealing groundbreaking findings that shed light on seizure mechanisms. Insights into cannabidiol (CBD) and the ketogenic diet as adjunctive therapies further broaden the spectrum of epilepsy management. Challenges in achieving seizure control with traditional therapies, including treatment resistance and individual variability, are addressed. The importance of staying updated with emerging trends in epilepsy management is emphasized, along with the hope for improved therapeutic options. Future research directions, such as combining therapies, AI applications, and non-invasive optogenetics, hold promise for personalized and effective epilepsy treatment. As the field advances, collaboration among researchers of natural and synthetic biochemistry, clinicians from different streams and various forms of medicine, and patients will drive progress toward better seizure control and a higher quality of life for individuals living with epilepsy.

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Section: Gene Therapies For Epilepsymentioning

confidence: 99%

A Comprehensive Review of Emerging Trends and Innovative Therapies in Epilepsy Management

Ghosh,

Sinha,

Ghosh

et al. 2023

Brain Sciences

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“…[1] ASOs were the first type of oligonucleotide to receive FDA approval and currently there are multiple drugs approved as well as in the FDA pipeline in this classification. [2] Other important classes of oligonucleotide therapeutics include small interfering RNA (siRNA), [3] CRISPR-based therapeutics, [4] and vaccines which can target cancer or infectious diseases. [5,6] An important property that all of these therapeutics have in common is that they must be chemically modified in order to survive in vivo and perform their desired functions.…”

Section: Introductionmentioning

confidence: 99%

Heterochiral modifications enhance robustness and function of DNA in living human cells

Mallette,

Lidke,

Lakin

2024

ChemBioChem

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Oligonucleotide therapeutics are becoming increasingly important as more are approved by the FDA, both for treatment and vaccination. Similarly, dynamic DNA nanotechnology is a promising technique that can be used to sense exogenous input molecules or endogenous biomarkers and integrate the results of multiple sensing reactions in situ via a programmed cascade of reactions. The combination of these two technologies could be highly impactful in biomedicine by enabling smart oligonucleotide therapeutics that can autonomously sense and respond to a disease state. A particular challenge, however, is the limited lifetime of standard nucleic acid components in living cells and organisms due to degradation by endogenous nucleases. In this work, we address this challenge by incorporating mirror‐image, L‐DNA nucleotides to produce heterochiral “gapmers”. We use dynamic DNA nanotechnology to show that these modifications keep the oligonucleotide intact in living human cells for longer than an unmodified strand. To this end, we used a sequential transfection protocol for delivering multiple nucleic acids into living human cells while providing enhanced confidence that subsequent interactions are actually occurring within the cells. Taken together, this work advances the state of the art of l‐nucleic acid protection of oligonucleotides and DNA circuitry for applications in vivo.

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A Revolutionary Approach for Combating Efflux Transporter-mediated Resistant Epilepsy: Advanced Drug Delivery Systems

Tonk,

Singh,

Sharma

et al. 2025

CPD

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Epilepsy is a persistent neurological condition that affects 60 million individuals globally, with recurrent spontaneous seizures affecting 80% of patients. Antiepileptic drugs (AEDs) are the main course of therapy for approximately 65% of epileptic patients, and the remaining 35% develop resistance to medication, which leads to Drug-Resistant Epilepsy (DRE). DRE continues to be an important challenge in clinical epileptology. There are several theories that attempt to explain the neurological causes of pharmacoresistance in epilepsy. The theory that has been studied the most is the transporter hypothesis. Therefore, it is believed that upregulation of multidrug efflux transporters at the blood-brain barrier (BBB), such as P-glycoprotein (P-gp), which extrudes AEDs from their target location, is the major cause, leading to pharmacoresistance in epilepsy. The most effective strategies for managing this DRE are peripheral and central inhibition of P-gp and maintaining an effective concentration of the drug in the brain parenchyma. Presently, no medicinal product that inhibits P-gp is being used in clinical practice. In this review, several innovative and promising treatment methods, including gene therapy, intracranial injections, Pgp inhibitors, nanocarriers, and precision medicine, are discussed. The primary goal of this work is to review the P-gp transporter, its substrates, and the latest novel treatment methods for the management of DRE.

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CRISPR: a tool with potential for genomic reprogramming in neurological disorders

Cited by 3 publications

References 100 publications

A Comprehensive Review of Emerging Trends and Innovative Therapies in Epilepsy Management

A Comprehensive Review of Emerging Trends and Innovative Therapies in Epilepsy Management

Heterochiral modifications enhance robustness and function of DNA in living human cells

A Revolutionary Approach for Combating Efflux Transporter-mediated Resistant Epilepsy: Advanced Drug Delivery Systems

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