Revolutionizing Treatment: Gene Therapy Offers Hope for Hurler Syndrome

Agarwal, Krisha

Permalink

https://hdl.handle.net/2142/126461 Copy

Description

Title

Revolutionizing Treatment: Gene Therapy Offers Hope for Hurler Syndrome

Author(s)

Agarwal, Krisha

Issue Date

2024

Keyword(s)

• Hurler Syndrome
• gene therapy
• lysosomal disorders

Date of Ingest

2025-03-13T21:24:14-05:00

Abstract

Hurler Syndrome, also known as mucopolysaccharidosis type I (MPS I), is a rare lysosomal disorder wherein genetic mutations prevent the synthesis of enzyme IDUA, disrupting the breakdown of sugar molecules. This autosomal recessive condition targets newborns and causes physical and cognitive abnormalities, potentially resulting in brain damage (Cleveland Clinic, 2022). Current treatments include bone marrow transplants, which are not only dangerous but also an unfavorable solution for progressive brain damage. Recently, a new form of gene therapy, Proprietary System (PS) gene editing, has shown promising results in mice as a treatment method, as concluded by researchers at the University of Minnesota. Using high-resolution resting-state functional MRI (rs-fMRI) technology, researchers could support normal neural connections using liver enzymes. This advanced approach also helps monitor brain connectivity in other lysosomal disorders affecting brain function (University of Minnesota, 2023).

Publisher

University of Illinois Undergraduate Neuroscience Society

Type of Resource

text

Genre of Resource

article

Language

eng

Copyright and License Information

Copyright 2024 Krisha Agarwal

Owning Collections