Gene Therapy and CRISPR Technologies: Revolutionizing Medicine

Tharushi Anargali Silva; Siavash Hosseinpour Chermahini; Pranav Balasubramonian; Kulsum Rahmath Alam; Vedant Malekar; Manthan Singh Jain; Nishaat Shaikh Faiz Ahmed

doi:doi:10.11648/j.mhs.20260204.12

Review Article |

Gene Therapy and CRISPR Technologies: Revolutionizing Medicine

Tharushi Anargali Silva^*, Siavash Hosseinpour Chermahini, Pranav Balasubramonian, Kulsum Rahmath Alam, Vedant Malekar, Manthan Singh Jain, Nishaat Shaikh Faiz Ahmed

Published in Medicine and Health Sciences (Volume 2, Issue 4)

Received: 17 May 2026 Accepted: 28 May 2026 Published: 6 November 2026

Views: Downloads:

Download PDF

Share This Article

Twitter
Linked In
Facebook

Abstract

Gene therapy and genome editing technologies have revolutionized modern medicine by allowing for the precise correction of genetic defects and novel therapeutic approaches for diseases that were previously considered untreatable. It is a technique that treats or prevents various diseases by replacing, correcting or inactivating faulty genes. Among these technologies, CRISPR-Cas systems have emerged as particularly simple, flexible, and rapidly expanding in their clinical applications. This review aims to synthesize the latest advances in gene therapy and CRISPR technologies, including mechanisms, delivery systems, therapeutic applications, clinical trials, challenges, and future perspectives. Recent developments such as base editing and prime editing have significantly improved the accuracy and safety of genome modification approaches. In addition, advances in viral and non-viral delivery systems have enhanced the efficiency of therapeutic gene transfer into target tissues. Current clinical trials have demonstrated promising outcomes in the treatment of inherited blood disorders, cancers, and metabolic diseases. The integration of artificial intelligence and bioinformatics tools is also improving guided RNA design and minimizing off-target effects in genome editing applications. Ethical considerations and regulatory frameworks remain essential for ensuring the safe and equitable implementation of these technologies in clinical practice. We conclude that despite the hurdles, high costs, long term safety monitoring, and immune system reactions to vectors, CRISPR-based strategies are on the verge of revolutionizing the practice of medicine with innovative, accurate solutions to hard hereditary, oncologic, and infectious diseases.

Published in	Medicine and Health Sciences (Volume 2, Issue 4)
DOI	10.11648/j.mhs.20260204.12
Page(s)	128-131
Creative Commons	This is an Open Access article, distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution and reproduction in any medium or format, provided the original work is properly cited.
Copyright	Copyright © The Author(s), 2026. Published by Science Publishing Group

Keywords

Gene Therapy, CRISPR-Cas Systems, Genome Editing, CRISPR-based Therapies

1. Introduction

Gene therapy and CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technologies have opened a new frontier in the field of precision medicine by allowing for the direct manipulation of the human genome for therapeutic ends. Gene therapies usually involves the introduction, deletion, or alteration of genetic material in a patient's cells to treat or prevent disease

[1]

. However, gene therapy lacked specificity in terms of inserting genes at desired locations in the genome. CRISPR systems, derived from the bacterial adaptive immunity system, enable the precise targeting and editing of DNA sequences using guide RNAs and Cas nucleases, making genome manipulation more accessible and efficient than previous technologies such as zinc-finger nucleases and TALENs

[4, 7]

. Recent reviews have emphasized that CRISPR technology has rapidly transformed biomedical research by providing efficient, accurate, and cost-effective genome editing tools for studying gene function and developing therapeutic strategies

[10]

. Thus, CRISPR technology has emerged as one of the most significant technologies in the field of research. It is being researched for its potential use in the treatment and cure of various diseases such as cancer and infections.

2. Historical Context and Mechanisms

2.1. Early Gene Therapy Strategies

Conventional gene therapy started with viral vectors that carried therapeutic genes into cells to supplement deficient ones

[2, 3]

. Of all the viruses used for gene therapy, adeno-associated virus and lentiviral vectors were found to be the most efficient vectors for gene therapy, showing relatively stable gene expression and lower toxicity when compared to earlier viruses used in gene therapy experiments. However, gene therapy experiments were also associated with some limitations. These limitations pointed to the need for genome editing technologies that are capable of making precise changes to DNA sequences in the genome. However, Adeno-associated virus (AAV) and lentiviral vectors have been the most popular because of their relatively benign integration patterns and efficient delivery

[8]

2.2. CRISPR-Cas Systems

CRISPR-Cas9 and other variants of Cas (e.g., Cas12a) brought about the ability to program genetic editing

[4, 7]

. This allows researchers to selectively target any region of the genome using specifically designed guide RNAs

[6]

. The Cas9 protein induces a double strand break in the targeted region, which is then repaired by the cell's machinery, allowing insertions, deletions, or corrections

[4, 6]

. Unlike previous genome editors, the CRISPR-Cas9 system has revolutionized genome editing through its simplicity of action. The main advantage of the CRISPR genome editors is their high programmability, as changing the DNA sequence requires only the modification of the 20-nucleotide RNA sequence of the guide RNA (sgRNA). In addition to genome editing, engineered CRISPR systems have been developed for transcriptional activation and gene regulation, enabling genome-scale control of gene expression and expanding the functional applications of CRISPR technologies beyond simple DNA cleavage

[9]

Technologies such as base editing and prime editing have improved the accuracy of editing by allowing the direct modification of individual nucleotides without inducing double-strand breaks, thus lowering the chances of off-target mutations

[16]

. These newer techniques represent an important step toward safer genome editing because they reduce the need for DNA breaks and allow more precise nucleotide modifications.

3. Delivery Systems for Therapeutic Editing

The efficient delivery of gene and CRISPR components is still a significant challenge

[18]

. The following are some of the most common methods used:

Viral Vectors: AAV and lentiviral vectors are still the most popular due to their high transduction efficiency

[1]

Non-viral Nanoparticles: Lipid nanoparticles and polymers are currently being developed for safe and repeated use

[11]

Physical Methods: Electroporation and microinjection allow precise targeting but lack scalability and translational potential

[18]

One of the biggest challenges in the clinical application of genome editing technologies is the efficient delivery of genome editing components into the body. The delivery process involves the transportation of large molecules such as the Cas nucleases and the guide RNA into the targeted cells, which is not an easy process due to the biological barriers that may be encountered in the process. However, the most commonly used delivery vehicles in genome editing technologies, especially the CRISPR technologies, are the viral vectors such as the adeno-associated virus and the lentivirus, which have the highest delivery efficiency compared to the non-viral delivery vehicles such as the lipid nanoparticles that can be used as safer delivery vehicles due to the possibility of repeated dosing

[18]

4. Clinical Applications

4.1. Genetic Disorders

Applications for CRISPR and gene therapy have rapidly progressed from proof-of-concept to clinical trials

[14]

. Clinical trials have shown significant efficacy in inherited disorders such as sickle cell disease and beta-thalassemia using edited stem cells

[5]

Recent studies have shown the efficacy of CRISPR editing in correcting specific mutations in metabolic and muscular disorders, thus proving its broad-spectrum potential

[12, 14]

. CRISPR technologies have shown promising applications in gene and cell therapy by enabling precise correction of disease-causing mutations and enhancing the development of next-generation therapeutic approaches

[13]

4.2. Oncology

CRISPR has shown immense potential in cancer therapy by improving immunotherapeutic approaches, including CAR-T cells engineered to improve tumor specificity and reduce immunosuppressive effects

[15, 17]

. CRISPR-mediated gene editing has also demonstrated potential in cancer therapy, including experimental strategies targeting hepatocellular carcinoma through modification of oncogenic pathways and tumor suppressor genes

[18]

4.3. Cardiovascular and Other Diseases

Preliminary clinical results have shown that CRISPR therapies can modulate genes responsible for lipid metabolism, thus providing hope for the treatment of cardiovascular diseases by permanently reducing harmful levels of cholesterol

[16]

4.4. Blood Disorders

Among the most advanced are those that target hemoglobinopathies and blood disorders, as there are several trials already ongoing

[5, 14]

5. Challenges and Ethical Implications

Despite the immense progress, the following challenges still exist:

Off-Target Effects: There is a possibility of unwanted modifications, which can lead to toxic mutations

[9]

Delivery Efficiency: The safe and precise delivery of the gene to different tissues is still a challenge

[18]

Ethical Implications: Germline editing has raised serious ethical questions regarding the long-term genetic modification of the human species, unequal access, and long-term implications

[9]

Regulatory Barriers: The differences in regulations across the globe make it challenging to adopt them on a larger scale

[16]

6. Future Outlook

The future of gene therapy and CRISPR technology will be shaped by:

Improved Precision Technology: The development of base and prime editors to overcome off-target issues

[18]

Expanded Therapeutic Indications: The application of CRISPR technology to more prevalent diseases, including common ones

[16]

AI Integration: The development of machine learning algorithms to design guide RNA and predict off-target sites, making it more efficient and safer

[16]

Ethical Guidelines: The development of proper ethical frameworks for its safe and effective use in the clinical setting

[9]

. Despite its transformative potential, CRISPR-based therapies still face challenges including off-target mutations, delivery efficiency, and long-term safety concerns that must be addressed before widespread clinical implementation

[11]

In addition to the technological advances in the field, the successful clinical application of CRISPR-based treatments will also depend on the improvement in the safety and regulation of the applications of genome editing technologies. The monitoring of the patients over time will be necessary in order to understand the potential risks involved in the unintended changes in the genes. Moreover, the ethical concerns surrounding the use of genome editing technologies, especially in the context of germline editing, will need to be addressed properly through guidelines and regulations at the international level. It is therefore necessary that there is continued collaboration and cooperation between scientists and policymakers in the effective application of technology in the management and treatment of human diseases

[11]

Abbreviations

CRISPR	Clustered Regularly Interspaced Short Palindromic Repeats
Cas	CRISPR-associated Protein
CRISPR-Cas9	CRISPR-associated Protein 9 System
sgRNA	Single-guide RNA
gRNA	Guide RNA
DNA	Deoxyribonucleic Acid
RNA	Ribonucleic Acid
AAV	Adeno-Associated Virus
CAR-T	Chimeric Antigen Receptor T-cell
TALENs	Transcription Activator-Like Effector Nucleases
ZFNs	Zinc Finger Nucleases
LNPs	Lipid Nanoparticles
DSB	Double-Strand Break
HDR	Homology-Directed Repair
NHEJ	Non-Homologous End Joining
AI	Artificial Intelligence
ML	Machine Learning
HCC	Hepatocellular Carcinoma
iPSCs	Induced Pluripotent Stem Cells
mRNA	Messenger RNA
ncRNA	Non-coding RNA
ex vivo	Outside the Body
in vivo	Inside the Body
FDA	Food and Drug Administration
WHO	World Health Organization
GMP	Good Manufacturing Practice

Acknowledgment

The authors extend their sincere appreciation to Prof. Siavash Hosseinpour Chermahini for his invaluable support and expert guidance, which greatly contributed to the successful completion of this research.

Author Contributions

Tharushi Anargali Silva: Conceptualization, Resources

Siavash Hosseinpour Chermahini: Supervision, Writing – review & editing

Pranav Balasubramonian: Data curation, Methodology

Kulsum Rahmath Alam: Data curation, Methodology

Vedant Malekar: Methodology, Writing – original draft

Manthan Singh Jain: Writing – review & editing

Nishaat Shaikh Faiz Ahmed: Conceptualization, Resources

Conflicts of Interest

The authors declare that there are no competing interests.

References

[1]	Park C-S, Habib O, Lee Y, Hur JK. Applications of CRISPR technologies to the development of gene and cell therapy. BMB Rep. 2024; 57(1): 2–11. https://pubmed.ncbi.nlm.nih.gov/38178651/
[2]	Mahmood T. CRISPR technology: Revolutionizing gene editing in the medical field. RMSR [Internet]. 2023 [cited 2026 Mar 5]; 1(02): 29–36.: https://medicalsciencereview.com/index.php/Journal/article/view/23
[3]	Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science [Internet].2012; 337(6096): 816–21. https://pmc.ncbi.nlm.nih.gov/articles/PMC6286148/
[4]	Frangoul H, Altshuler D, Cappellini MD, Chen Y-S, Domm J, Eustace BK, et al. CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. N Engl J Med [Internet]. 2021; 384(3): 252–60 https://pubmed.ncbi.nlm.nih.gov/33283989/
[5]	Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex genome engineering using CRISPR/Cas systems. Science [Internet]. 2013; 339(6121): 819–23. https://pubmed.ncbi.nlm.nih.gov/23287718/
[6]	Doudna JA, Charpentier E. Genome editing. The new frontier of genome engineering with CRISPR-Cas9. Science [Internet]. 2014; 346(6213): 1258096. https://pubmed.ncbi.nlm.nih.gov/25430774/
[7]	Konermann S, Brigham MD, Trevino AE, Joung J, Abudayyeh OO, Barcena C, et al. Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex. Nature [Internet]. 2015; 517(7536): 583–8. https://pubmed.ncbi.nlm.nih.gov/25494202/
[8]	Karimi MA, Paryan M, Behrouzian Fard G, Sadeghian H, Zarrinfar H, Hosseini Bafghi M. Challenges and opportunities in the application of CRISPR-Cas9: A review on genomic editing and therapeutic potentials. Med Princ Pract [Internet]. 2026; 35(1): 1–17. https://pmc.ncbi.nlm.nih.gov/articles/PMC12503735/
[9]	(N.d.). Mdpi.com. Retrieved March 5, 2026, from https://www.mdpi.com/1422-0067/25/24/13321
[10]	Zhang, M.-L., Li, H.-B., & Jin, Y. (2024). Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy. Frontiers in Genetics, 15, 1364742. https://www.frontiersin.org/journals/genetics/articles/10.3389/fgene.2024.1364742/full
[11]	Laurent, M., Geoffroy, M., Pavani, G., & Guiraud, S. (2024). CRISPR-based gene therapies: From preclinical to clinical treatments. Cells (Basel, Switzerland), 13(10), 800. https://www.mdpi.com/2073-4409/13/10/800
[12]	Wang, S.-W., Gao, C., Zheng, Y.-M., Yi, L., Lu, J.-C., Huang, X.-Y., Cai, J.-B., Zhang, P.-F., Cui, Y.-H., & Ke, A.-W. (2022). Current applications and future perspective of CRISPR/Cas9 gene editing in cancer. Molecular Cancer, 21(1), 57. https://pubmed.ncbi.nlm.nih.gov/35189910/
[13]	Iyer KA, Tenchov R, Lotti Diaz LM, Jain P, Thite T, Deng Y, Zhou QA. CRISPR technology: transforming the future of medicine and diagnostics. Biochemistry. 2025; 64(24): 4628‑60 https://pubmed.ncbi.nlm.nih.gov/41284414/
[14]	Maganti, H. B., Kirkham, A. M., Bailey, A. J. M., Shorr, R., Kekre, N., Pineault, N., & Allan, D. S. (2022). Use of CRISPR/Cas9 gene editing to improve chimeric antigen-receptor T cell therapy: A systematic review and meta-analysis of preclinical studies. Cytotherapy, 24(4), 405–412. https://pubmed.ncbi.nlm.nih.gov/35039239/
[15]	Amjad, E., Pezzani, R., & Sokouti, B. (2024). A review of the literature on the use of CRISPR/Cas9 gene therapy to treat hepatocellular carcinoma. Oncology Research, 32(3), 439–461. https://pubmed.ncbi.nlm.nih.gov/38361756/
[16]	Lee, H., Rho, W.-Y., Kim, Y.-H., Chang, H., & Jun, B.-H. (2025). CRISPR-Cas9 gene therapy: Non-viral delivery and stimuli-responsive nanoformulations. Molecules (Basel, Switzerland), 30(3), 542. https://pubmed.ncbi.nlm.nih.gov/39942646/
[17]	Ma, Y., & Qi, L. S. (2024). The realization of CRISPR gene therapy. Nature Chemical Biology, 20(7), 791–795. https://pubmed.ncbi.nlm.nih.gov/38872012/
[18]	Chira S, Nutu A, Isacescu E, Bica C, Pop L, Ciocan C, Berindan-Neagoe I. Genome editing approaches with CRISPR/Cas9 for cancer treatment: critical appraisal of preclinical and clinical utility, challenges, and future research. Cells. 2022; 11(17): 2781. Available from: https://pubmed.ncbi.nlm.nih.gov/36139356/

Cite This Article

Plain Text BibTeX RIS

APA Style

Silva, T. A., Chermahini, S. H., Balasubramonian, P., Alam, K. R., Malekar, V., et al. (2026). Gene Therapy and CRISPR Technologies: Revolutionizing Medicine. Medicine and Health Sciences, 2(4), 128-131. https://doi.org/10.11648/j.mhs.20260204.12

Copy | Download

ACS Style

Silva, T. A.; Chermahini, S. H.; Balasubramonian, P.; Alam, K. R.; Malekar, V., et al. Gene Therapy and CRISPR Technologies: Revolutionizing Medicine. Med. Health Sci. 2026, 2(4), 128-131. doi: 10.11648/j.mhs.20260204.12

Copy | Download

AMA Style

Silva TA, Chermahini SH, Balasubramonian P, Alam KR, Malekar V, et al. Gene Therapy and CRISPR Technologies: Revolutionizing Medicine. Med Health Sci. 2026;2(4):128-131. doi: 10.11648/j.mhs.20260204.12

Copy | Download

@article{10.11648/j.mhs.20260204.12,
  author = {Tharushi Anargali Silva and Siavash Hosseinpour Chermahini and Pranav Balasubramonian and Kulsum Rahmath Alam and Vedant Malekar and Manthan Singh Jain and Nishaat Shaikh Faiz Ahmed},
  title = {Gene Therapy and CRISPR Technologies: Revolutionizing Medicine},
  journal = {Medicine and Health Sciences},
  volume = {2},
  number = {4},
  pages = {128-131},
  doi = {10.11648/j.mhs.20260204.12},
  url = {https://doi.org/10.11648/j.mhs.20260204.12},
  eprint = {https://article.sciencepublishinggroup.com/pdf/10.11648.j.mhs.20260204.12},
  abstract = {Gene therapy and genome editing technologies have revolutionized modern medicine by allowing for the precise correction of genetic defects and novel therapeutic approaches for diseases that were previously considered untreatable. It is a technique that treats or prevents various diseases by replacing, correcting or inactivating faulty genes. Among these technologies, CRISPR-Cas systems have emerged as particularly simple, flexible, and rapidly expanding in their clinical applications. This review aims to synthesize the latest advances in gene therapy and CRISPR technologies, including mechanisms, delivery systems, therapeutic applications, clinical trials, challenges, and future perspectives. Recent developments such as base editing and prime editing have significantly improved the accuracy and safety of genome modification approaches. In addition, advances in viral and non-viral delivery systems have enhanced the efficiency of therapeutic gene transfer into target tissues. Current clinical trials have demonstrated promising outcomes in the treatment of inherited blood disorders, cancers, and metabolic diseases. The integration of artificial intelligence and bioinformatics tools is also improving guided RNA design and minimizing off-target effects in genome editing applications. Ethical considerations and regulatory frameworks remain essential for ensuring the safe and equitable implementation of these technologies in clinical practice. We conclude that despite the hurdles, high costs, long term safety monitoring, and immune system reactions to vectors, CRISPR-based strategies are on the verge of revolutionizing the practice of medicine with innovative, accurate solutions to hard hereditary, oncologic, and infectious diseases.},
 year = {2026}
}

Copy | Download

TY  - JOUR
T1  - Gene Therapy and CRISPR Technologies: Revolutionizing Medicine
AU  - Tharushi Anargali Silva
AU  - Siavash Hosseinpour Chermahini
AU  - Pranav Balasubramonian
AU  - Kulsum Rahmath Alam
AU  - Vedant Malekar
AU  - Manthan Singh Jain
AU  - Nishaat Shaikh Faiz Ahmed
Y1  - 2026/11/06
PY  - 2026
N1  - https://doi.org/10.11648/j.mhs.20260204.12
DO  - 10.11648/j.mhs.20260204.12
T2  - Medicine and Health Sciences
JF  - Medicine and Health Sciences
JO  - Medicine and Health Sciences
SP  - 128
EP  - 131
PB  - Science Publishing Group
SN  - 3070-6300
UR  - https://doi.org/10.11648/j.mhs.20260204.12
AB  - Gene therapy and genome editing technologies have revolutionized modern medicine by allowing for the precise correction of genetic defects and novel therapeutic approaches for diseases that were previously considered untreatable. It is a technique that treats or prevents various diseases by replacing, correcting or inactivating faulty genes. Among these technologies, CRISPR-Cas systems have emerged as particularly simple, flexible, and rapidly expanding in their clinical applications. This review aims to synthesize the latest advances in gene therapy and CRISPR technologies, including mechanisms, delivery systems, therapeutic applications, clinical trials, challenges, and future perspectives. Recent developments such as base editing and prime editing have significantly improved the accuracy and safety of genome modification approaches. In addition, advances in viral and non-viral delivery systems have enhanced the efficiency of therapeutic gene transfer into target tissues. Current clinical trials have demonstrated promising outcomes in the treatment of inherited blood disorders, cancers, and metabolic diseases. The integration of artificial intelligence and bioinformatics tools is also improving guided RNA design and minimizing off-target effects in genome editing applications. Ethical considerations and regulatory frameworks remain essential for ensuring the safe and equitable implementation of these technologies in clinical practice. We conclude that despite the hurdles, high costs, long term safety monitoring, and immune system reactions to vectors, CRISPR-based strategies are on the verge of revolutionizing the practice of medicine with innovative, accurate solutions to hard hereditary, oncologic, and infectious diseases.
VL  - 2
IS  - 4
ER  -

Copy | Download

Author Information

Tharushi Anargali Silva

International Medical School, Alte University, Tbilisi, Georgia

Contact Email
Siavash Hosseinpour Chermahini

School of Science and Technology, The University of Georgia, Tbilisi, Georgia
Pranav Balasubramonian

International Medical School, Alte University, Tbilisi, Georgia
Kulsum Rahmath Alam

International Medical School, Alte University, Tbilisi, Georgia
Vedant Malekar

International Medical School, Alte University, Tbilisi, Georgia
Manthan Singh Jain

International Medical School, Alte University, Tbilisi, Georgia
Nishaat Shaikh Faiz Ahmed

International Medical School, Alte University, Tbilisi, Georgia

Download PDF

Submit an Article

Sections

Plain Text BibTeX RIS

APA Style

Silva, T. A., Chermahini, S. H., Balasubramonian, P., Alam, K. R., Malekar, V., et al. (2026). Gene Therapy and CRISPR Technologies: Revolutionizing Medicine. Medicine and Health Sciences, 2(4), 128-131. https://doi.org/10.11648/j.mhs.20260204.12

Copy | Download

ACS Style

Silva, T. A.; Chermahini, S. H.; Balasubramonian, P.; Alam, K. R.; Malekar, V., et al. Gene Therapy and CRISPR Technologies: Revolutionizing Medicine. Med. Health Sci. 2026, 2(4), 128-131. doi: 10.11648/j.mhs.20260204.12

Copy | Download

AMA Style

Silva TA, Chermahini SH, Balasubramonian P, Alam KR, Malekar V, et al. Gene Therapy and CRISPR Technologies: Revolutionizing Medicine. Med Health Sci. 2026;2(4):128-131. doi: 10.11648/j.mhs.20260204.12

Copy | Download

@article{10.11648/j.mhs.20260204.12,
  author = {Tharushi Anargali Silva and Siavash Hosseinpour Chermahini and Pranav Balasubramonian and Kulsum Rahmath Alam and Vedant Malekar and Manthan Singh Jain and Nishaat Shaikh Faiz Ahmed},
  title = {Gene Therapy and CRISPR Technologies: Revolutionizing Medicine},
  journal = {Medicine and Health Sciences},
  volume = {2},
  number = {4},
  pages = {128-131},
  doi = {10.11648/j.mhs.20260204.12},
  url = {https://doi.org/10.11648/j.mhs.20260204.12},
  eprint = {https://article.sciencepublishinggroup.com/pdf/10.11648.j.mhs.20260204.12},
  abstract = {Gene therapy and genome editing technologies have revolutionized modern medicine by allowing for the precise correction of genetic defects and novel therapeutic approaches for diseases that were previously considered untreatable. It is a technique that treats or prevents various diseases by replacing, correcting or inactivating faulty genes. Among these technologies, CRISPR-Cas systems have emerged as particularly simple, flexible, and rapidly expanding in their clinical applications. This review aims to synthesize the latest advances in gene therapy and CRISPR technologies, including mechanisms, delivery systems, therapeutic applications, clinical trials, challenges, and future perspectives. Recent developments such as base editing and prime editing have significantly improved the accuracy and safety of genome modification approaches. In addition, advances in viral and non-viral delivery systems have enhanced the efficiency of therapeutic gene transfer into target tissues. Current clinical trials have demonstrated promising outcomes in the treatment of inherited blood disorders, cancers, and metabolic diseases. The integration of artificial intelligence and bioinformatics tools is also improving guided RNA design and minimizing off-target effects in genome editing applications. Ethical considerations and regulatory frameworks remain essential for ensuring the safe and equitable implementation of these technologies in clinical practice. We conclude that despite the hurdles, high costs, long term safety monitoring, and immune system reactions to vectors, CRISPR-based strategies are on the verge of revolutionizing the practice of medicine with innovative, accurate solutions to hard hereditary, oncologic, and infectious diseases.},
 year = {2026}
}

Copy | Download

TY  - JOUR
T1  - Gene Therapy and CRISPR Technologies: Revolutionizing Medicine
AU  - Tharushi Anargali Silva
AU  - Siavash Hosseinpour Chermahini
AU  - Pranav Balasubramonian
AU  - Kulsum Rahmath Alam
AU  - Vedant Malekar
AU  - Manthan Singh Jain
AU  - Nishaat Shaikh Faiz Ahmed
Y1  - 2026/11/06
PY  - 2026
N1  - https://doi.org/10.11648/j.mhs.20260204.12
DO  - 10.11648/j.mhs.20260204.12
T2  - Medicine and Health Sciences
JF  - Medicine and Health Sciences
JO  - Medicine and Health Sciences
SP  - 128
EP  - 131
PB  - Science Publishing Group
SN  - 3070-6300
UR  - https://doi.org/10.11648/j.mhs.20260204.12
AB  - Gene therapy and genome editing technologies have revolutionized modern medicine by allowing for the precise correction of genetic defects and novel therapeutic approaches for diseases that were previously considered untreatable. It is a technique that treats or prevents various diseases by replacing, correcting or inactivating faulty genes. Among these technologies, CRISPR-Cas systems have emerged as particularly simple, flexible, and rapidly expanding in their clinical applications. This review aims to synthesize the latest advances in gene therapy and CRISPR technologies, including mechanisms, delivery systems, therapeutic applications, clinical trials, challenges, and future perspectives. Recent developments such as base editing and prime editing have significantly improved the accuracy and safety of genome modification approaches. In addition, advances in viral and non-viral delivery systems have enhanced the efficiency of therapeutic gene transfer into target tissues. Current clinical trials have demonstrated promising outcomes in the treatment of inherited blood disorders, cancers, and metabolic diseases. The integration of artificial intelligence and bioinformatics tools is also improving guided RNA design and minimizing off-target effects in genome editing applications. Ethical considerations and regulatory frameworks remain essential for ensuring the safe and equitable implementation of these technologies in clinical practice. We conclude that despite the hurdles, high costs, long term safety monitoring, and immune system reactions to vectors, CRISPR-based strategies are on the verge of revolutionizing the practice of medicine with innovative, accurate solutions to hard hereditary, oncologic, and infectious diseases.
VL  - 2
IS  - 4
ER  -

Copy | Download