Page 6 - 2020 Winter CMTA Report

CMTA Funds

Cutting-Edge

Gene Editing

Studies for

Type 2 CMT

rs. Bruce Conklin and Luke Drs. Conklin and Judge, lead- ment of research tools and poten-
Judge of the Gladstone ers in genome editing, and their tial disease treatments. Editing the
Institutes and UCSF team (www.ConklinLab.org) have genomes of rodents and other
Departments of Medicine already made substantial progress organisms that scientists com-
Dand Pediatrics will use a in testing CRISPR in stem cell monly study has brought forward
$653,000 grant from the CMTA models of CMT2A and CMT2E. numerous discoveries about how
in 2020 to develop the gene-edit- The San Francisco Bay the genome is con-
ing technique known as CRISPR area is at the forefront The studies nected to physical traits
for application to CMT2A, 2E of technology develop- like eye color and
and 2F. ment in the field: In have the diseases like CMT.
Gene editing is a group of 2012, Dr. Jennifer potential to CRISPR can
technologies that give researchers Doudna and her team make deletions in the
and scientists the ability to modify at Berkeley discovered advance genome and/or be
DNA. Genetic material can be how CRISPR could be gene editing engineered to insert
added, removed or altered at cer- used for DNA editing. technology new DNA sequences.
tain locations in the genome, Dr. Doudna now The CRISPR system
much like cutting and pasting directs the Innovative for CMT. was adapted from a
information in a Word document. Genomics Institute naturally occurring
Methods to modify DNA in the (IGI), where Dr. Con- gene editing systems
genome have been around for klin serves as deputy director in bacteria. The bacteria capture
more than 30 years, but CRISPR (https://innovativegenomics.org/). snippets of DNA from invading
technology has brought major The newly funded project will viruses and use them to create
improvements in the speed, cost, focus on developing the pre-clini- DNA segments known as
accuracy and efficiency of gene cal tools and preliminary data CRISPR arrays. The CRISPR
editing. needed to take the next steps for arrays allow the bacteria to
eventually test- “remember” the viruses so that if
ing CRISPR they attack again, the bacteria can
therapies in target their DNA. Remarkably,
human clinical this bacterial defense system works
trials. in human cells to edit DNA and
The history perhaps treat genetic diseases.
of gene editing While many leading scientists
technologies believe that the use of CRISPR
shows the in clinical trials is still years away,
remarkable the studies have the potential to
progress in this advance this technology for CMT
field and the and set a new standard for thera-
critical role that peutic engineering for motor
basic science neuron disease that can be
The Conklin Lab Team research plays in extended to other neurological
the develop- diseases. h

6 THE CMTA REPORT WINTER 2020