Jul 2026· International Journal of Drug Delivery Technology· 0 citations· 3 references
TL;DR
The potential application of CRISPR technology for the possible management of geneticbased conditions, including sickle-cell anemia, β-thalassemia, cystic fibrosis, and Duchenne muscular dystrophy is described.
Abstract
Background
CRISPR-Cas9 technology has become a revolutionary tool in molecular biology and has transformed the
landscape of genetic research and therapeutics. CRISPR has allowed for an unprecedented opportunity to target,
modify and correct disease-causing mutations at the source, given the rapid, efficient and precise modifications
of these DNA sequences. The simplicity and versatility of CRISPR, especially in comparison to previous gene
editing platforms such as zinc-finger nucleases and TALENs, is appealing to today's researchers and clinicians,
making CRISPR a preferred gene-modifying strategy for laboratory studies, and a pathway for future clinical
trials.
Scope
This review describes the potential application of CRISPR technology for the possible management of geneticbased conditions. This review will focus on monogenic disorders, including sickle-cell anemia, β-thalassemia,
cystic fibrosis, and Duchenne muscular dystrophy. Strategies using CRISPR are currently being evaluated in in
vivo approaches and ex vivo approaches using patient-derived cells. The advantages of CRISPR, including
precision, specificity, and potential cure, are compared with the disadvantages of traditional gene therapy,
including inefficiency, costs, and immune difficulties.
Challenges
Possible problems with CRISPR technology and applications also remain. Off-target effects and incomplete
edits are based on safety issues/concerns for long-term consequences. Bioethics issues, including germline
editing and possible uses for enhancement of non-therapeutic applications, require even stricter oversight and
regulation.
This review systematically summarizes the developmental logic, core mechanisms, clinical applications, advantages and limitations of the three generations of CRISPR-Cas technology in monogenic disorders, and analyzes the key challenges such as delivery efficiency, long-term safety, and treatment accessibility.
Yiwen Wang· Theoretical and Natural Scie...· 0 citations
The CRISPR-Cas9 genome-editing system has emerged as a promising technology for the precise correction of disease-causing mutations and has shown significant potential in treating various genetic disorders. However, the successful clinical application of CRISPR-Cas9 largely depends on the development of safe and efficient delivery systems. Among the available approaches, nanoparticle-mediated delivery has gained considerable attention for its low immunogenicity, high biocompatibility, improved targeting, and capacity to deliver diverse CRISPR cargoes. This review summarises the current strategies of nanoparticle-mediated CRISPR-Cas9 delivery, including lipid, polymeric, hybrid, inorganic, and biomimetic nanoparticles. It also discusses major pharmacological challenges such as off-target effects, biodistribution, endosomal escape, immunogenicity, toxicity, and regulatory considerations. Furthermore, recent therapeutic applications in genetic disorders, including sickle cell disease, β-thalassemia, Duchenne muscular dystrophy, cystic fibrosis, hereditary transthyretin amyloidosis, and cancer, are highlighted. Finally, future perspectives on advanced nanocarrier design and clinical translation are discussed. Nanoparticle-mediated CRISPR-Cas9 delivery holds great promise for advancing precision medicine and developing effective therapies for genetic disorders.
Shouvik Mondal, Kriti Kumari· Advanced International Journ...· 0 citations
Colorectal cancer (CRC) is a leading cause of cancer-related deaths worldwide, characterized by genetic heterogeneity and the accumulation of mutations in key oncogenes and tumor suppressor genes. CRISPR-Cas9 technology has greatly advanced genetic research by enabling precise genome editing. This review focuses on the innovative applications of CRISPR-Cas9 in CRC research, particularly its role in identifying novel therapeutic targets, elucidating mechanisms of drug resistance, and uncovering metabolic and stem cell pathway alterations in tumorigenesis. We highlight the diverse CRISPR systems, including Cas9, Cas12, Cas13, and advanced variants such as CRISPR activation (CRISPRa), CRISPR interference (CRISPRi), base editing, and prime editing, which have expanded gene knockout studies and enhanced our understanding of CRC. Despite these breakthroughs, challenges such as off-target effects and delivery limitations remain. Ongoing efforts to refine CRISPR technology aim to enhance its precision and clinical applicability, ultimately paving the way for more effective and personalized treatment strategies for CRC. In this review, we explore these advances and focus on the latest developments in CRISPR-based approaches for CRC treatment.
Mohadeseh Hassannia, P. Amirifar· Cancer Treatment and Researc...· 0 citations
CRISPR has emerged as a next-generation gene-editing tool with the potential to target the molecular pathways associated with ageing and related disorders. It functions through RNA-guided Cas nucleases, directing DNA cleavage and utilizing the native DNA repair machinery for genetic manipulations. Advances in CRISPR technology have significantly enhanced the precision and flexibility of techniques for genome editing. The enzyme Cas9's ability to cut DNA at exact site has revolutionized genome editing by enabling accurate modifications within living eukaryotic cells. This review critically examines recent developments in CRISPR-based technologies, including Cas9, Cas12, base editing, prime editing, and CRISPR-mediated gene regulation. It highlights their rising applications in ageing research, with more emphasis on neurodegenerative disorders such as Alzheimer's and Parkinson's diseases. The review also discusses the major pharmacological and translational challenges that currently limit clinical applications, including inefficient tissue-specific delivery, off-target genome editing, immunogenicity, manufacturing complexity, and long-term safety concerns. Also, recent progress in both, viral and non-viral delivery methods are critically evaluated, including adeno-associated viruses, lentivirus vectors, lipid nanoparticles, gold nanoparticles, exosomes, electroporation, and microinjection, is thoroughly discussed to highlight their therapeutic potential and translational limitations. Current studies indicate that CRISPR-based approaches have preclinical potential for targeting important hallmarks of ageing, particularly genomic instability, telomere attrition, and mitochondrial dysfunction. Other hallmarks of ageing, such as stem cell exhaustion, epigenetic modifications, and microbiome changes, are at earlier stages of development. Overall, this review describes future strategies for developing safe, precise, and clinically translatable CRISPR-based treatments to promote healthy ageing.
Sakshi Rathore, Akash Gupta, Kamal Shah et al.· Ageing Research Reviews· 0 citations
CRISPR-based genome editing has transformed the therapeutic possibilities of genomic medicine by enabling programmable modification of disease-associated genes and regulatory pathways. This review analyzed the evolution, current clinical applications, emerging therapeutic strategies, safety limitations, ethical challenges, and future perspectives of CRISPR-based technologies within an international framework that incorporates Latin America. An integrative literature review was conducted using scientific evidence published between 2012 and July 2026, with emphasis on peer-reviewed studies indexed in major biomedical databases and relevant international and regulatory documents. The evidence showed a rapid progression from foundational CRISPR-Cas9 research toward base editing, prime editing, ex vivo cellular modification, and direct in vivo genome editing. Clinical translation was most advanced in hematology, particularly in sickle cell disease and transfusion-dependent β-thalassemia, where genome-edited autologous hematopoietic stem and progenitor cells have demonstrated clinically meaningful therapeutic outcomes. Applications in oncology and hereditary transthyretin amyloidosis further demonstrated the feasibility of engineered immune cells and systemic in vivo genome editing. However, broader clinical implementation remains limited by delivery and tissue targeting, off-target and unintended on-target modifications, immunogenicity, manufacturing complexity, long-term safety, regulatory requirements, and accessibility. The international analysis identified heterogeneous capacity for clinical implementation, emphasizing the need to strengthen genomic infrastructure, population representation, professional training, regulatory preparedness, and research participation in Latin America. CRISPR has therefore progressed from an experimental genome-engineering technology to a clinically relevant therapeutic platform, although its future impact will depend on the ability to combine molecular precision and durable therapeutic benefit with rigorous safety assessment, responsible governance, and equitable access across diverse populations and healthcare systems.
Gabriel Alejandro, Ortega Moreno, G. Amaya et al.· International science journa...· 0 citations
Gene therapy is evolving from gene addition to precise genome editing, enabling the direct correction of disease-causing mutations. Breakthrough technologies, such as clustered regularly interspaced short palindromic repeats-CRISPR-associated protein (CRISPR-Cas) nucleases, base editors, prime editors, and CRISPR-associated transposases are reshaping the therapeutic landscape. This review covers the progression of precision editing technologies and their clinical applications, spanning from ex vivo therapies to in vivo treatments targeting vital organs. The rise of personalized medicine, highlighted by therapies, such as carbamoyl phosphate synthetase 1 editing, underscores the shift toward N-of-1 medicine for rare diseases. Clinical trial progress, delivery and accessibility challenges, and the role of AI in optimizing editing tools and predicting outcomes are also discussed. These innovations are transforming genetic medicine, offering the promise of safer, more durable, and personalized cures.
Tongtong Cui, Bojin Li, Bingyu Cai et al.· Trends in Molecular Medicine· 0 citations