Thematic Report

Crispr 2.0

by Shaun Cochran / Feb 26, 2019

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When Crispr was first invented seven years ago, the global scientific community marvelled at its simultaneous simplicity and profundity. This chemical process was simple enough to operate within bacteria, but flexible enough to offer far-reaching utility in entire organic systems.

Therapeutic, diagnostic and agricultural applications
Subsequent years of research have unveiled that Crispr, while groundbreaking, comes with the all-too-often-discovered limitations, unintended consequences and uncertainties. Not just one chemical tool that allows us to invoke the genetic editing process, it is actually a portfolio of enzymes that is still growing as new discoveries are made. Better still, the limitations and strengths of each enzyme are often different, allowing for a rich tapestry of medical challenges and solutions to come together.

Commercial applications in agriculture
Genome editing is driving a generation of new traits in crops and livestock. Crispr-edited plants can resist diseases, improve yields and thrive under harsher conditions, while edited livestock can be made more muscular or have horns genetically removed for easier handling. However, exciting as this field is, the regulatory landscape is still evolving. Beyond agriculture, Crispr could increase biofuel yields, making alternative energy sources more economically viable.

Commercial ex vivo and in vivo applications
Crispr shows great promise in treating genetic disease. Human therapeutics can be split into two categories: ex vivo (outside the human body) and in vivo (inside). Ex vivo approaches include using Crispr to edit patient’s stem cells or T-cells then transplanting them back in to fight blood disorders like sickle-cell disease or cancer. In vivo approaches include delivering Crispr components to the lungs or muscles to tackle cystic fibrosis or muscular dystrophy. A number of strong therapy candidates will enter human trials soon.

Crispr therapy development challenges and improvements
Despite enormous progress, challenges remain. Before these technologies can be delivered to patients, safety concerns and Crispr-technique issues must be addressed, such as off-target editing, genomic rearrangements and immune responses. Also, not all genetic diseases can currently be targeted. Crispr still has low-efficiency in many cells and it can be difficult to deliver the genome-editing machinery in patients. However, these hurdles are creating pockets for new research and innovation. Crispr research is moving at breakneck pace. Protein engineering of Crispr enzymes has generated variants that are more specific or flexible, and these alternatives are enabling the targeting of more genome sites, even different types of nucleic acids, such as RNA.

What’s next?
Crispr continues to expand into unimaginable applications, including engineering pig organs for human transplants; rapid, direct evolution of proteins; automated and high-throughput gene editing; and screening for the identification of drug targets. Future discoveries of different Crispr enzymes with unexpected functionalities will drive the field in novel and exciting directions.

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