Keynote Presentation: Derya Unutmaz, MD, The Jackson Laboratories, CT “Exploring the limits of AI in Medicine”

1. Regulation of lysosomal function by nanotechnology

2. Genetically modified macrophages targeting inflamed brains in animal models of Parkinson disease

3. Lysosome dysfunction as an emerging nanomaterial toxicity Blood-brain barrier delivery for

lysosomal disorders with IgG-lysosomal enzyme fusion proteins

4. Lysosomal therapies and drug delivery strategies: Liposomes and nanoparticles

 Organoids and Lab-Grown Models: Patient-derived organoids (mini-organs) replicate lysosomal

dysfunction, offering platforms for testing therapies and studying disease mechanisms.

1. Modeling neurological disease with human brain organoids

2. Fabry disease models using kidney organoid and spheroid systems

3. Modeling neuronopathic Lysosomal disorders with patient-derived culture systems

4. Brain organoid modeling of Gaucher disease

5. Modeling Fabry disease cardiomyopathy using engineered heart tissue and organoids

 Theranostics in Lysosomal Disorders Theranostics is an innovative medical field that combines

diagnostic and therapeutic strategies into a single platform, enabling precision medicine tailored

to individual patients.

1. Molecular conjugate probes and MRI-guided Gene therapy

2. Small molecule probes to interrogate organ involvement

3. Visualization of cholesterol deposition in lysosomes in NPC disease

4. Potential Targeting Mechanisms using GAGS for Bone-Directed Therapies

5. CRISPR Base editing of the GLB1 gene is therapeutic in GM1 gangliosidosis patient-derived cells

6. The sustained delivery of mRNA lipid nanoparticles to cartilage using microcarriers in MPS

7. The delivery of mRNa to the muscle for the treatment of Pompe disease

1. Biomarkers for Gene Therapy Clinical Trials of Lysosomal Disorders

2. AAV-mediated gene therapy for sialidosis.

3. Patient Preferences for Gene Therapy in Lysosomal Disorders

4. CNS effect of systemic administration of AAV Gene Transfer Therapies: Lessons from MPS IIIA

5. Co-administration of GT and UCBT for Krabbe disease

6. Gene replacement therapy for MPS3C

7. Lentiviral Gene Therapy for Lysosomal Disorders

1. Opportunities and challenges for deep learning in cell dynamics research.

2. An Open-Label Clinical Trial Using a Second-Generation Artificial-Intelligence-Based Therapeutic

Regimen in Patients with Gaucher Disease Treated with Enzyme Replacement Therapy

3. An AI-based approach and pandas-mic platform to identify lysosomal cystine export regulating

mTORC1 signaling

4. Assessment of bone involvement in Gaucher disease: An AI-based approach