AI in Gene Therapy| Molecular Pharmaceutics|Unit-5

AI in Gene Therapy | Molecular Pharmaceutics | Unit-5
Artificial Intelligence (AI) is revolutionizing the field of gene therapy by accelerating the discovery, design, and delivery of genetic medicines. In molecular pharmaceutics, AI tools are applied to analyze large genomic datasets, identify disease-causing mutations, and predict effective gene-editing strategies such as CRISPR-Cas systems. AI-driven algorithms help in optimizing vector design, improving target specificity, minimizing off-target effects, and enhancing the safety and efficacy of gene therapies. Moreover, machine learning models assist in patient selection, treatment response prediction, and personalized therapy development. By integrating AI with molecular pharmaceutics, researchers can significantly reduce development time, cost, and failure rates, making gene therapy more precise and accessible.

1. Savitribai Phule Pune University , Pune A seminar on Artificial Intelligence in Gene Therapy Bhujbal knowledge city, MET’s Institute Of Pharmacy, Adgaon , Nashik.2024-2025. Guided by : DR.N.A THOMBRE D epartment of Pharmaceutics NAME OF STUDENT Rohit Gangadhar Chaudhari M.Pharm First Year( Sem I I )

2. R&D Cell, MET's Institute of Pharmacy, Nashik Content Introduction of AI Gene Theraphy Overview Role of AI in Gene Therapy Role of AI in Gene Therapy Design Role of AI in Gene Therapy Delivery Applications AI Tools in Gene Therapy Benefits of AI in Gene Therapy Challenges and Limitations Future Directions Conclusion References

3. What is Artificial Intelligence(AI)? R&D Cell, MET's Institute of Pharmacy, Nashik AI is the simulation of human intelligence by machines. Includes Machine Learning (ML), Deep Learning (DL), and Natural Language Processing (NLP ). Basically AI Refers to   the ability of computer systems to perform tasks that typically require human intelligence, such as learning, reasoning, problem-solving, and decision-making Example AI Tools:1.Chatgpt 2.Perplexity AI 3.Sci-Space 4.NotebookLM

4. Gene Therapy Overview Gene therapy is an experimental technique that uses genes to treat or prevent disease, aiming to treat disorders by inserting a gene into a patient's cells instead of using drugs or surgery. It's a promising treatment for inherited disorders, certain cancers, and viral infections. Genetic material needs a carrier, called a vector , to deliver the gene into cells. A)Viral Vectors( AAV,Lentivirus ) B)Non-Viral Vectors( liposomes,nanoparticles ) Types of Gene Therapy: R&D Cell, MET's Institute of Pharmacy, Nashik

5. R&D Cell, MET's Institute of Pharmacy, Nashik 1.Somatic cell gene therapy is a technique that involves the transfer of a gene to a diseased somatic cell. This transfer can occur either within the body ( in vivo ) or outside the body ( ex vivo ). Both viral and non-viral gene therapy vectors are utilized for this purpose. 2. Ex-Vivo Gene Therapy   a type of gene therapy where cells are modified outside the body (in a laboratory setting) and then transplanted back into the patient. 3.In -Vivo Gene Therapy is   a method of treating diseases by directly delivering genetic material into cells within the patient's body. 4 . In Situ Gene therapy involves delivering therapeutic genes directly to the precise location where they are needed within the body. 

6. Artificial Intelligence (AI) is revolutionizing gene therapy by enhancing the design and delivery of treatments, leading to more precise, efficient, and personalized therapies. AI tools, particularly machine learning and deep learning, are being used to analyze vast datasets, identify gene targets, optimize gene editing techniques like CRISPR-Cas9, and improve gene delivery systems R&D Cell, MET's Institute of Pharmacy, Nashik Role Of AI in Gene Therapy

7. Role of AI in Gene Therapy Design Gene Target Identification: AI analyzes genomic databases to find faulty genes. Predicts disease-gene relationships (e.g., CRISPR gene editing targets ). Sequence Design: AI optimizes guide RNAs (gRNAs) in CRISPR. Prevents off-target effects . Mutation Prediction: AI forecasts how mutations affect protein function . R&D Cell, MET's Institute of Pharmacy, Nashik

8. Role of AI in Gene Therapy Delivery Vector Selection and Engineering: AI predicts optimal delivery vectors based on tissue type and gene payload. Designs synthetic vectors (AAV capsids) for better targeting. Personalized Delivery: AI models individual genetic profiles for tailored therapy. Biodistribution Modeling: Predicts how vectors will spread and express genes in the body. R&D Cell, MET's Institute of Pharmacy, Nashik

9. Applications of AI Tools in Gene Therapy R&D Cell, MET's Institute of Pharmacy, Nashik

10. Benefits Of AI in Gene Therapy R&D Cell, MET's Institute of Pharmacy, Nashik Accurate gene target prediction . Faster therapy design Reduced cost and lab workload Personalized treatment planning Minimized adverse effects Efficient vector development

11. Challenges and Limitations R&D Cell, MET's Institute of Pharmacy, Nashik

12. R&D Cell, MET's Institute of Pharmacy, Nashik Smarter and Safer Vectors: AI will continue to help design novel viral and non-viral vectors with higher efficiency and better safety profiles. Real Time Monitoring: Real Time monitoring of gene expression using AI Integrated Healthcare: AI will integrate multi-omics, imaging, and clinical data to provide clinicians with comprehensive, data-driven insights for personalized care. Advanced Bio-robotics: AI will support the design and control of microscopic " nanobots " capable of autonomous, targeted drug and gene delivery. Future Directions

13. Conclusion AI is transforming the landscape of gene therapy from design to delivery. Improves efficacy , safety, and personalization. Still evolving – requires collaborative efforts between biologists, data scientists, and clinicians . When we use AI (Artificial Intelligence) and gene therapy together, it's like having a super-smart helper. This partnership gives us the best chance to finally beat many diseases that are caused by faulty genes and create a healthier future for everyone." R&D Cell, MET's Institute of Pharmacy, Nashik

14. References Hasanzadeh A, Hamblin MR, Kiani J, Noori H, Hardie JM, Karimi M, et al. Could artificial intelligence revolutionize the development of nanovectors for gene therapy and mRNA vaccines? Nano Today. 2022;47:101665. Dixit S, Kumar A, Srinivasan K, Vincent PMDR, Ramu Krishnan N. Advancing genome editing with artificial intelligence: opportunities, challenges, and future directions. Front Bioeng Biotechnol . 2024;11:1335901. Erdoğan S. Integration of Artificial Intelligence and Genome Editing System for Determining the Treatment of Genetic Disorders. Balkan Med J. 2024;41(6):419-20. Vilhekar RS, Rawekar A. Artificial Intelligence in Genetics. Cureus . 2024;16(1):e52035. Kitaoka Y, Uchihashi T, Kawata S, Nishiura A, Yamamoto T, Hiraoka S- i , et al. Role and Potential of Artificial Intelligence in Biomarker Discovery and Development of Treatment Strategies for Amyotrophic Lateral Sclerosis. Int J Mol Sci. 2025;26(9):4346. National Human Genome Research Institute. CRISPR [Internet]. National Institutes of Health; 2023 [cited 2025 Jul 19]. Available from: https:// www.genome.gov/genetics-glossary/CRISPR Samraik M. Researchers use machine learning to improve gene therapy [Internet]. Stanford Report; 2025 Jun 3 [cited 2025 Jul 20]. Available from: https:// news.stanford.edu/stories/2025/06/machine-learning-ai-cell-gene-therapies AZoLifeSciences . AI-Driven Protein Engineering for Gene Therapy Advancements [Internet]. AZoLifeSciences ; 2025 Apr 23 [cited 2025 Jul 20]. Available from: https://www.azolifesciences.com/news/20250423/AI-Powered-Protein-Engineering-for-Precise-and-Scalable-Gene-Therapy.aspx Form Bio. The Role of AI in Gene Therapy [Internet]. Form Bio; 2024 Mar 20 [cited 2025 Jul 20]. Available from: https://www.formbio.com/blog/ai-gene-therapy   R&D Cell, MET's Institute of Pharmacy, Nashik

15. R&D Cell, MET's Institute of Pharmacy, Nashik