@TheHilaaluk asked: "if we have a wild type sequence that we wish to insert, how do we know that that particular wild type will be curative considering that wild types may differ based on ethnicity/populations? i.e. a wt sequence for one population may not be the wt for another population". Our expert's response: If there is ethnic genetic heterogeneity, and this is a totally bespoke therapy, I’d choose the WT gene from the parent (unlikely situation). If it’s not bespoke, I’d choose the sequence found at highest rates in the population with the highest amount of disease burden to insert. However, I do doubt it would really matter as many of these genes and their variance to create multiple normal ‘wild types’ are unlikely to have a significant phenotypic effect if replaced with other WTs.
TIMESTAMPS 00:00 Introductions and talk overview 01:45 History and the present of gene editing 05:24 What is gene editing? How does gene editing work (in detail)? 10:59 The ZFN and TALEN gene editing systems 14:40 About the CRISPR/Cas system 21:50 CRISPR in practice 28:59 CRISPR use cases: from drug discovery to as a drug itself 35:10 CRISPR/Cas limitations 38:23 CRISPR's future uses: as a nickase or epigenome modifier 42:39 CRISPR in the clinic 45:45 CRISPR's economics and CRISPR ethics 49:40 Q&A and wrap-up
@TheHilaaluk asked: "if we have a wild type sequence that we wish to insert, how do we know that that particular wild type will be curative considering that wild types may differ based on ethnicity/populations? i.e. a wt sequence for one population may not be the wt for another population".
Our expert's response: If there is ethnic genetic heterogeneity, and this is a totally bespoke therapy, I’d choose the WT gene from the parent (unlikely situation). If it’s not bespoke, I’d choose the sequence found at highest rates in the population with the highest amount of disease burden to insert. However, I do doubt it would really matter as many of these genes and their variance to create multiple normal ‘wild types’ are unlikely to have a significant phenotypic effect if replaced with other WTs.
TIMESTAMPS
00:00 Introductions and talk overview
01:45 History and the present of gene editing
05:24 What is gene editing? How does gene editing work (in detail)?
10:59 The ZFN and TALEN gene editing systems
14:40 About the CRISPR/Cas system
21:50 CRISPR in practice
28:59 CRISPR use cases: from drug discovery to as a drug itself
35:10 CRISPR/Cas limitations
38:23 CRISPR's future uses: as a nickase or epigenome modifier
42:39 CRISPR in the clinic
45:45 CRISPR's economics and CRISPR ethics
49:40 Q&A and wrap-up