07 - Luigi Naldini, MD, PhD - Giants of Gene Therapy
May 30, 2023
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In this enlightening conversation, Luigi Naldini, a pioneer in gene therapy and a professor at San Raffaele University, shares his remarkable journey from Torino to groundbreaking research. He discusses the innovative use of lentiviral vectors and their potential to cure genetic disorders and cancer. Naldini addresses challenges in making gene therapies accessible in low-income countries and emphasizes the importance of diverse representation in genomic research. He also offers insights for aspiring scientists and explores the joy of balancing work with personal passions like art and music.
Dr. Luigi Naldini's pioneering work on lentiviral vectors has fundamentally transformed gene therapy by improving gene transfer efficiency and clinical outcomes.
The establishment of the San Raffaele Telethon Institute exemplifies Dr. Naldini's commitment to bridging scientific research with practical medical applications for gene therapy.
Deep dives
Pioneering Advances in Lentiviral Vector-Based Gene Therapy
Dr. Louis G. Naldini is renowned for his pioneering work in lentiviral vector-based gene therapy, which has significantly influenced clinical trials for various genetic diseases and cancer. His innovations have enhanced the efficiency of gene transfer, particularly in non-dividing cells, by leveraging the natural mechanisms of the HIV virus. Naldini's groundbreaking design included self-inactivating vectors, which mitigated genotoxic risks associated with earlier gene transfer methods. This research not only improved therapeutic outcomes but also laid a foundation for safe and effective gene therapies used in clinical settings today.
Transition from Research to Clinical Application
Dr. Naldini's transition from academia to the clinical environment was marked by the establishment of the San Raffaele Telethon Institute in Milan, Italy. This institute was designed to bridge the gap between scientific research and practical medical applications, promoting collaboration between clinicians and scientists. Under his leadership, the institute has grown to employ approximately 250 people and has been involved in many successful gene therapy trials. This unique setting fosters translational science, enabling rapid advancement from laboratory discoveries to clinical implementation.
Challenges and Future Directions in Gene Therapy
Despite the successes of lentiviral vectors, challenges remain, particularly regarding the inherent genotoxicity risks associated with random insertion into the genome. The need for more targeted insertion methods has propelled research into gene editing technologies, which might complement or even eventually replace some applications of lentiviral vectors. Moreover, the rising costs and accessibility issues in gene therapy emphasize the importance of developing more affordable treatments, especially for underserved populations with diseases like sickle cell disease. Naldini advocates for innovative delivery methods and improved accessibility to ensure that these groundbreaking therapies reach a broader patient demographic.
Mentorship and the Future of Gene Therapy
As a mentor, Dr. Naldini emphasizes the importance of enthusiasm and a commitment to both fundamental and applied science in the next generation of researchers. He encourages young scientists to balance innovation with a realistic understanding of the translational potential of their work, ensuring that their projects align with clinical needs. With ongoing advancements in RNA technology and more, he believes that the future of gene therapy lies in creating smarter disease treatments, particularly through intelligent design in gene editing and therapy delivery systems. His vision includes leveraging the combined strengths of gene therapy and editing to enhance patient outcomes and expand therapeutic possibilities.
Luigi Naldini, MD, PhD, discusses his intercontinental career and the future of gene editing and use of lentiviral vectors during a one-on-one conversation with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the seventh episode of Giants of Gene Therapy!
Dr. Naldini is a Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. He has achieved worldwide renown as the pioneer of lentivirus gene therapy, a tool that offers hope for cures to deadly diseases.
Dr. Naldini has been a mentor to many young scientists and a prolific researcher with more than 280 publications. He has founded three biotech startups. He’s spoken in more than 150 congresses worldwide. And he’s received numerous global awards, including the Grand Officer of the Order for Merit of the Italian Republic (one of the highest-ranking honors in Italy), the Louis-Jeantet Prize for Medicine in Switzerland, and Outstanding Achievement Awards from both the American Society of Gene and Cell Therapy and European Society of Gene and Cell Therapy.
Dr. Naldini has been an ASGCT member since 2000, the year he also attended the 3rd ASGCT Annual Meeting. He’s served on the Society’s board and committees.