#323 - CRISPR and the future of gene editing: scientific advances, genetic therapies, disease treatment potential, and ethical considerations | Feng Zhang, Ph.D.

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Oct 28, 2024

Feng Zhang, a groundbreaking professor of neuroscience at MIT and a leader in gene editing, discusses his pioneering work with CRISPR technology. He explores the revolutionary journey of CRISPR, from its discovery to its potential in treating genetic diseases like sickle cell anemia. Zhang also addresses the ethical debates surrounding gene editing, particularly concerning germline modifications. He shares insights from his personal journey, the impact of mentorship, and the future potential of genetic medicine, highlighting both challenges and innovative solutions in the field.

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INSIGHT

CRISPR Delivery to Liver Cells

Liver cells are effectively targeted in vivo with CRISPR using lipid nanoparticles, similar to mRNA vaccine delivery.
This approach shows promise for treating genetic liver diseases and potentially cardiovascular disease.

ANECDOTE

CRISPR for Eye Diseases

CRISPR gene therapy shows promise for treating genetic eye diseases like LCA10 by inactivating mutant genes.
However, vision restoration is limited by retinal degeneration and delivery inefficiencies.

INSIGHT

Cost of CRISPR Therapies

The high cost of CRISPR therapies is primarily due to development and scaling of manufacturing processes.
Large-scale production of mRNA and nanoparticles for human treatment is a key challenge.

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Feng Zhang, a professor of neuroscience at MIT and a pioneering figure in gene editing, joins Peter to discuss his groundbreaking work in CRISPR technology, as well as his early contributions to optogenetics. In this episode, they explore the origins of CRISPR and the revolutionary advancements that have transformed the field of gene editing. Feng delves into the practical applications of CRISPR for treating genetic diseases, the importance of delivery methods, and the current successes and challenges in targeting cells specific tissues such as those in the liver and eye. He also covers the ethical implications of gene editing, including the debate around germline modification, as well as reflections on Feng's personal journey, the impact of mentorship, and the future potential of genetic medicine.

We discuss:

Feng's background, experience in developing optogenetics, and his shift toward improving gene-editing technologies [2:45];
The discovery of CRISPR in bacterial DNA and the realization that these sequences could be harnessed for gene editing [10:45];
How the CRISPR system fights off viral infections and the role of the Cas9 enzyme and PAM sequence [21:00];
The limitations of earlier gene-editing technologies prior to CRISPR [28:15];
How CRISPR revolutionized the field of gene editing, potential applications, and ongoing challenges [36:45];
CRISPR's potential in treating genetic diseases and the challenges of effective delivery [48:00];
How CRISPR is used to treat sickle cell anemia [53:15];
Gene editing with base editing, the role of AI in protein engineering, and challenges of delivery to the right cells [1:00:15];
How CRISPR is advancing scientific research by fast-tracking the development of transgenic mice [1:06:45];
Advantages of Cas13's ability to direct CRISPR to cleave RNA and the advances and remaining challenges of delivery [1:11:00];
CRISPR-Cas9: therapeutic applications in the liver and the eye [1:19:45];
The ethical implications of gene editing, the debate around germline modification, regulation, and more [1:30:45];
Genetic engineering to enhance human traits: challenges, trade-offs, and ethical concerns [1:40:45];
Feng's early life, the influence of the American education system, and the critical role teachers played in shaping his desire to explore gene-editing technology [1:46:00];
Feng's optimism about the trajectory of science [1:58:15]; and
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