Precise editing of pathogenic nucleotide repeat expansions in iPSCs using paired prime editor

HY Hwang, D Gim, H Yi, H Jung, J Lee… - Nucleic Acids …, 2024 - academic.oup.com
Nucleotide repeat expansion disorders, a group of genetic diseases characterized by the
expansion of specific DNA sequences, pose significant challenges to treatment and therapy …

Blocking the dimerization of polyglutamine-expanded androgen receptor protects cells from DHT-induced toxicity by increasing AR turnover

A Lisberg, Y Liu, DE Merry - Journal of Biological Chemistry, 2024 - ASBMB
Spinal and bulbar muscular atrophy (SBMA) is a neuromuscular degenerative disease
caused by a polyglutamine expansion in the androgen receptor (AR). This mutation causes …

A phenotypically robust model of spinal and bulbar muscular atrophy in Drosophila

K Richardson, M Sengupta, A Sujkowski… - Journal of …, 2024 - Wiley Online Library
Spinal and bulbar muscular atrophy (SBMA) is an X‐linked disorder that affects males who
inherit the androgen receptor (AR) gene with an abnormal CAG triplet repeat expansion …

Chronic Exercise and the Treatment of Progressive Disorders

K Richardson - 2023 - search.proquest.com
Exercise is a known, beneficial treatment for a variety of normal (aging) and abnormal
(disease) declines in health. These benefits can be observed in a range of tissues, including …

Identifying the Mechanism Behind the Role of the AR N/C Interaction in SBMA

A Lisberg - 2024 - search.proquest.com
Spinal bulbar muscular atrophy (SBMA) is an X-linked, progressive neuromuscular
degenerative disease affecting approximately 1 in 50000 males worldwide. Patients display …

ASSESSING SELECTIVE ANDROGEN RECEPTOR MODULATORS AS POTENTIAL THERAPUETICS

A Lisberg, DE Merry - Acceptable as a Thesis Fulfilling Part of …, 2024 - search.proquest.com
Spinal bulbar muscular atrophy (SBMA) is an X-linked neuromuscular degenerative disease
affecting approximately 1 in 50,000 males worldwide. There is currently no effective …