Molecular Genetics and Metabolism

GM2 gangliosidosis insights: Tay-Sachs models via CRISPR-Cas9

Pub Date : 2024-02-01 DOI: 10.1016/j.ymgme.2023.108052 Diego Suarez, Angela J. Espejo, C. Alméciga-Díaz

A long-term observation of ophthalmology assessment in atypical CLN2 patients receiving ICV cerliponase alfa treatment

Pub Date : 2024-02-01 DOI: 10.1016/j.ymgme.2023.107910 Audrey Kao, Harriet Chang, Rahul Bhola, Raymond Y. Wang

Novel approach for tracheal resection in Morquio syndrome type A with end-stage critical airway obstruction: A UK case series

Pub Date : 2024-02-01 DOI: 10.1016/j.ymgme.2023.107914 J. Kenth, Elizabeth Maughan, Colin Butler, Simon Jones, Richard Hewitt