PKD Life - Summer 2023 - 22

DR_MICROBE/GETTY IMAGES. INSETS: UT SOUTHWESTERN
MEDICAL CENTER.
THE RESEARCH REPORT
The Promise
of Gene
Therapy
Preliminary research
suggests that harnessing
PKD1 or PKD2 may stop the
progression of ADPKD.
By Debra Gordon
R esearchers have known for decades
that a mutation in the PKD1 or PKD2
genes prevents the gene from producing
polycystin-1 (PC1), a protein required for
healthy kidney function. Without it, the
kidneys develop autosomal dominant
polycystic kidney disease, or ADPKD. Yet,
as the name implies, just one copy of the
gene is mutated; the other (we have two
copies of every gene) is healthy. So, why
doesn't that healthy gene pick up the
slack and increase its production of PC1
to prevent or at least slow disease progression
in ADPKD?
Vishal Patel, M.D.
BREAKING DOWN
THE BARRICADES
It's not for lack of trying,
say Vishal Patel, M.D., and
Ronak Lakhia, M.D., of the
University of Texas Southwestern
in Dallas. The protein-producing
process starts
with messenger RNA, or
mRNA. It carries the instructions
from the DNA and is
translated to produce the
protein. The problem has to
do with the body's quest for
homeostasis. If the healthy
22 PKD LIFE * SUMMER 2023

PKD Life - Summer 2023

Table of Contents for the Digital Edition of PKD Life - Summer 2023

Contents
PKD Life - Summer 2023 - Cover1
PKD Life - Summer 2023 - Cover2
PKD Life - Summer 2023 - Contents
PKD Life - Summer 2023 - 2
PKD Life - Summer 2023 - 3
PKD Life - Summer 2023 - 4
PKD Life - Summer 2023 - 5
PKD Life - Summer 2023 - 6
PKD Life - Summer 2023 - 7
PKD Life - Summer 2023 - 8
PKD Life - Summer 2023 - 9
PKD Life - Summer 2023 - 10
PKD Life - Summer 2023 - 11
PKD Life - Summer 2023 - 12
PKD Life - Summer 2023 - 13
PKD Life - Summer 2023 - 14
PKD Life - Summer 2023 - 15
PKD Life - Summer 2023 - 16
PKD Life - Summer 2023 - 17
PKD Life - Summer 2023 - 18
PKD Life - Summer 2023 - 19
PKD Life - Summer 2023 - 20
PKD Life - Summer 2023 - 21
PKD Life - Summer 2023 - 22
PKD Life - Summer 2023 - 23
PKD Life - Summer 2023 - 24
PKD Life - Summer 2023 - 25
PKD Life - Summer 2023 - 26
PKD Life - Summer 2023 - 27
PKD Life - Summer 2023 - 28
PKD Life - Summer 2023 - Cover3
PKD Life - Summer 2023 - Cover4
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2024fall
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2024winterspring
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2023summer
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2022fall
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2022summer
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2022spring
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2021fall
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2021summer
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2020fall
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2020summer
https://www.nxtbook.com/nxtbooks/pkdlife/pkdlife_2020spring
https://www.nxtbookmedia.com