Technologies of gene editing and related clinical trials for the treatment of genetic and acquired diseases: a systematic review DOI Creative Commons
Wessam E. Sharaf‐Eldin

Egyptian Journal of Medical Human Genetics, Journal Year: 2024, Volume and Issue: 25(1)

Published: March 7, 2024

Abstract Background Gene editing can produce irreversible permanent changes to the genetic material at predetermined sequences, avoiding random integration, which is major drawback of classical gene therapy. The technology has invaded all approaches engineering and biotechnology with versatile applications in agriculture, industry, medicine. Main body present review displays different mechanisms editing. Special emphasis been given therapeutic where registered clinical trials have addressed. Islamic ethical concerns also highlighted. Conclusion great advantages technology, coupled splendid efforts scientists develop systems superior efficacy safety would provide an effective avenue for treating a wide range human diseases near future.

Language: Английский

Curcumin-Based Nanomedicines in the Treatment of Inflammatory and Immunomodulated Diseases: An Evidence-Based Comprehensive Review DOI Creative Commons
Lucas Fornari Laurindo,

Gabriel Magno de Carvalho,

Bárbara de Oliveira Zanuso

et al.

Pharmaceutics, Journal Year: 2023, Volume and Issue: 15(1), P. 229 - 229

Published: Jan. 10, 2023

Curcumin (CUR) is a polyphenol extracted from the rhizome of Curcuma longa that possesses potent anti-inflammatory and antioxidant potential. Despite CUR’s numerous beneficial effects on human health, it has limitations, such as poor absorption. Nano-based drug delivery systems have recently been applied to improve solubility bioavailability potentialize its health effects. This review investigated different CUR-based nanomedicines inflammatory immunomodulated diseases. PUBMED, EMBASE, COCHRANE, GOOGLE SCHOLAR databases were searched, Scale for Assessment Narrative Review Articles (SANRA) was used quality assessment PRISMA guidelines. Overall, 66 studies included comprising atherosclerosis, rheumatoid arthritis (RA), Alzheimer’s disease (AD), Parkinson’s (PD), multiple sclerosis (MS), Huntington’s (HD), bowel diseases (IBD), psoriasis, liver fibrosis, epilepsy, COVID-19. The available scientific show there are many known nanoformulations with curcumin. They can be found in nanosuspensions, nanoparticles, nanoemulsions, solid lipid particles, nanocapsules, nanospheres, liposomes. These formulations CUR effectively adjuvants several immune-mediated atheroma plaque formation, RA, dementia, AD, PD, MS, IBD, COVID-19, anti-fibrotic fibrotic disease.

Language: Английский

Citations

53
Synergistic strategies for glioblastoma treatment: CRISPR-based multigene editing combined with immune checkpoint blockade DOI Creative Commons
Xiaolin Liu, Xiao Liu, Xiaonan Luo

et al.

Journal of Nanobiotechnology, Journal Year: 2025, Volume and Issue: 23(1)

Published: Feb. 7, 2025

Language: Английский

Citations

4
CRISPR/Cas9 Landscape: Current State and Future Perspectives DOI Open Access
Marina A. Tyumentseva, Marina A. Tyumentseva, В. Г. Акимкин

et al.

International Journal of Molecular Sciences, Journal Year: 2023, Volume and Issue: 24(22), P. 16077 - 16077

Published: Nov. 8, 2023

CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 is a unique genome editing tool that can be easily used in wide range of applications, including functional genomics, transcriptomics, epigenetics, biotechnology, plant engineering, livestock breeding, gene therapy, diagnostics, and so on. This review focused on the current CRISPR/Cas9 landscape, e.g., Cas9 variants with improved properties, Cas9-derived fusion proteins, delivery methods, pre-existing immunity against anti-CRISPR their possible roles function improvement. Moreover, this presents detailed outline CRISPR/Cas9-based diagnostics therapeutic approaches. Finally, addresses future expansion editors’ toolbox orthologs other CRISPR/Cas proteins.

Language: Английский

Citations

34
Exploring molecular mechanisms, therapeutic strategies, and clinical manifestations of Huntington’s disease DOI
Alaa Shafie, Amal Adnan Ashour,

Saleha Anwar

et al.

Archives of Pharmacal Research, Journal Year: 2024, Volume and Issue: 47(6), P. 571 - 595

Published: May 19, 2024

Language: Английский

Citations

9
A review on molecular scissoring with CRISPR/Cas9 genome editing technology DOI

Muskan Irfan,

Hammad Majeed, Tehreema Iftikhar

et al.

Toxicology Research, Journal Year: 2024, Volume and Issue: 13(4)

Published: July 1, 2024

Genome editing is a technology to make specific changes in the DNA of cell or an organism. It has significantly altered landscape life sciences, facilitating establishment exceedingly customized genetic modifications. Among various genome technologies, CRISPR/Cas9 system, endonuclease induces double stranded break and enabling modifications genome, surfaced as formidable adaptable instrument. Its significance cannot be overstated, it not only allows for manipulation genomes model organisms but also holds great potential revolutionary advances medicine, particularly treating diseases. This review paper explores remarkable journey CRISPR/Cas9, its natural function, mechanisms, transformative impact on finally use artificial intelligence other intelligent manufacturing tools used. The introduction provides background editing, emphasizing emergence CRISPR/Cas9. Subsequent sections comprehensively elucidate disease modeling, agriculture, biotechnology, address therapeutic applications, ongoing clinical trials while discussing prospects ethical implications. We summarized key findings, indicating that empowered creation disease-specific animal models. invaluable insights into pathogenic mechanisms opens new avenues drug discovery, reaffirming editing. Finally we discussed importance continued research collaboration comprehensive utilization inherent capabilities this molecular precision tool shaping forthcoming advancements.

Language: Английский

Citations

9
Unraveling the Puzzle of Therapeutic Peptides: A Promising Frontier in Huntington’s Disease Treatment DOI
Shakir Ahamad,

Nargis Bano,

Sameera Khan

et al.

Journal of Medicinal Chemistry, Journal Year: 2024, Volume and Issue: 67(2), P. 783 - 815

Published: Jan. 11, 2024

Huntington's disease (HD) is a neurodegenerative genetic disorder characterized by mutation in the huntingtin (HTT) gene, resulting production of mutant protein (mHTT). The accumulation mHTT leads to development toxic aggregates neurons, causing cell dysfunction and, eventually, death. Peptide therapeutics target various aspects HD pathology, including reduction and aggregation inhibition, extended CAG mRNA degradation, modulation dysregulated signaling pathways, such as BDNF/TrkB signaling. In addition, these peptide also detrimental interactions with InsP3R1, CaM, or Caspase-6 proteins mitigate HD. This Perspective provides detailed perspective on anti-HD therapeutic peptides, highlighting their design, structural characteristics, neuroprotective effects, specific mechanisms action. for exhibit promise preclinical models, but further investigation required confirm effectiveness viable strategies, recognizing that no approved therapy currently exists.

Language: Английский

Citations

8
Unexplored power of CRISPR-Cas9 in neuroscience, a multi-OMICs review DOI
Mohammad Banazadeh, Ardavan Abiri,

Mohammad Mahdi Poortaheri

et al.

International Journal of Biological Macromolecules, Journal Year: 2024, Volume and Issue: 263, P. 130413 - 130413

Published: Feb. 24, 2024

Language: Английский

Citations

8
A Review of the Common Neurodegenerative Disorders: Current Therapeutic Approaches and the Potential Role of Bioactive Peptides DOI
Kuldeep Singh, Jeetendra Kumar Gupta, Shivendra Kumar

et al.

Current Protein and Peptide Science, Journal Year: 2024, Volume and Issue: 25(7), P. 507 - 526

Published: April 2, 2024

Abstract:: Neurodegenerative disorders, which include Alzheimer's disease (AD), Parkinson's (PD), Huntington's (HD), and amyotrophic lateral sclerosis (ALS), represent a significant growing global health challenge. Current therapies predominantly focus on symptom management rather than altering progression. In this review, we discuss the major therapeutic strategies in practice for these highlighting their limitations. For AD, mainstay treatments are cholinesterase inhibitors N-methyl-D-aspartate (NMDA) receptor antagonists. PD, dopamine replacement therapies, including levodopa, commonly used. HD is managed primarily with symptomatic treatments, reusable extends survival ALS. However, none of halts or substantially slows neurodegenerative process. contrast, review highlights emerging research into bioactive peptides as potential agents. These naturally occurring synthetically designed molecules can interact specific cellular targets, potentially modulating processes. Preclinical studies suggest that may mitigate oxidative stress, inflammation, protein misfolding, common pathological features diseases. Clinical trials using neurodegeneration limited but show promising initial results. instance, hemiacetal, γ-secretase inhibitor peptide, has shown AD by reducing amyloid-beta production, though its development was discontinued due to side effects. Despite advancements, many challenges remain, identifying optimal peptides, confirming mechanisms action, overcoming obstacles related delivery brain. Future should prioritize discovery novel improve our understanding pharmacokinetics pharmacodynamics. Ultimately, approach lead more effective moving beyond modify course devastating

Language: Английский

Citations

7
Therapeutic approaches targeting aging and cellular senescence in Huntington's disease DOI Creative Commons
Asif Ahmad Bhat, Ehssan Moglad, Muhammad Afzal

et al.

CNS Neuroscience & Therapeutics, Journal Year: 2024, Volume and Issue: 30(10)

Published: Oct. 1, 2024

Huntington's disease (HD) is a devastating neurodegenerative that manifested by gradual loss of physical, cognitive, and mental abilities. As the advances, age has major impact on pathogenic signature mutant huntingtin (mHTT) protein aggregation. This review aims to explore intricate relationship between aging, mHTT toxicity, cellular senescence in HD. Scientific data interplay mHTT, HD were collected from several academic databases, including PubMed, Google Scholar, Google, ScienceDirect. The search terms employed "AGING," "HUNTINGTON'S DISEASE," "MUTANT HUNTINGTIN," "CELLULAR SENESCENCE." Additionally, gather information molecular mechanisms potential therapeutic targets, was extended include relevant such as "DNA DAMAGE," "OXIDATIVE STRESS," "AUTOPHAGY." According research, aging leads worsening pathophysiology through some processes. result accumulation, promoted, which causes DNA damage, oxidative stress, decreased autophagy, increased inflammatory responses. Pro-inflammatory cytokines other substances are released senescent cells, may worsen neuronal damage course disease. It been shown treatments directed at these pathways reduce symptoms enhance longevity experimental animals, pointing new possibility treating condition. Through their amplification harmful effects play crucial roles development Comprehending interplays creates novel opportunities for measures targeted alleviating enhancing patients' quality life.

Language: Английский

Citations

7
Landscape of NRXN1 Gene Variants in Phenotypic Manifestations of Autism Spectrum Disorder: A Systematic Review DOI Open Access
Jaimee Cooper,

Jeenu Mittal,

Akhila Sangadi

et al.

Journal of Clinical Medicine, Journal Year: 2024, Volume and Issue: 13(7), P. 2067 - 2067

Published: April 2, 2024

Background: Autism spectrum disorder (ASD) is a complex neurodevelopmental condition characterized by social communication challenges and repetitive behaviors. Recent research has increasingly focused on the genetic underpinnings of ASD, with Neurexin 1 (NRXN1) gene emerging as key player. This comprehensive systematic review elucidates contribution NRXN1 variants in pathophysiology ASD. Methods: The protocol for this was designed priori registered PROSPERO database (CRD42023450418). A risk bias analysis conducted using Joanna Briggs Institute (JBI) critical appraisal tool. We examined various studies that link disruptions discussing both genotypic variability resulting phenotypic expressions. Results: Within review, there marked heterogeneity observed ASD manifestations among individuals mutations. presence mutations population emphasizes gene’s role synaptic function neural connectivity. Conclusion: not only highlights but also need further to unravel disorder. better knowledge about multifaceted can provide crucial insights into neurobiological foundations autism pave way novel therapeutic strategies.

Language: Английский

Citations

6