Christian Mueller

This paper describes an AAV-mediated gene therapy strategy for GBA1-PD and Gaucher Disease, demonstrating robust GCase replenishment and cross-correction in preclinical models.

The paper develops a machine learning model to predict AAV capsid fitness from amino acid sequences, achieving high accuracy and generalizability across datasets.

This study develops an AAV vector for ARSA gene replacement in MLD, showing improved brain biodistribution and therapeutic effects in mice and non-human primates.

This study shows focused ultrasound enhances gene delivery to deep brain structures when recombinant adeno-associated virus is administered in the cerebrospinal fluid.

This study removes a hexanucleotide repeat expansion in C9ORF72 using CRISPR/Cas9 in cell and animal models, reducing disease hallmarks.

The paper identifies the CHOP-c-JUN complex as a key driver of liver proteotoxicity in AAT deficiency by enhancing SERPINA1 transcription and ATZ accumulation.