AI in Medicine: Transforming the Landscape of Tissue-Based DIagnostics
Keynote Speaker: Behzad Najafinam, MD
Day 1
1. Nanotechnology.
08:00
Arrival/Breakfast
08:00
09:00 – 9:40
Keynote Lecture
09:40 – 10:00
Blood-brain barrier delivery for lysosomal storage disorders with IgG-lysosomal enzyme fusion proteins.
09:40 – 10:00
10:20 – 10:40
Lysosomal therapies and drug delivery strategies: Liposomes and nanoparticles.
10:40 - 11:00
Muscle-directed precision RNA delivery platforms: Centryin-Based Nano delivery in Pompe disease
10:40 - 11:00
11:00 – 11:30
Q&A
11:30 – 11:50
Coffee break
11:30 – 11:50
2. Organoids and Lab-Grown Models.
11:50 – 12:10
Modeling neuronopathic storage disorders with patient-derived culture systems.
11:50 – 12:10
12:10 – 12:30
Evaluation of microglia from GBA1 iPSC Cell lines.
12:30 – 12:50
Engineered heart tissue and organoids for inherited cardiac diseases (Fabry disease).
12:30 – 12:50
12:50 – 01:10
Generation of a brain organoid platform to study neuronopathic Gaucher disease and therapeutic strategies.
1:10 - 1:40
Q&A
1:10 - 1:40
1:40 - 2:50
Lunch
2:20 - 2:50
Selected poster presentations.
2:20 - 2:50
3. Theranostics in Lysosomal Disorders.
02:50 – 03:10
Exploring fluorescent techniques to analyze the morphology, positioning, and function of the lysosome.
02:50 – 03:10
03:10 – 03:30
Mitochondrial and lysosomal dynamics by fluorescent microscopy.
03:30 – 03:50
Targeting mechanisms using GAGs for bone-directed therapies.
03:30 – 03:50
03:50 – 04:10
The next generation of gene-editing modalities is emerging as a new drug development field in LDs—the application of prime editing as a therapeutic strategy in Rare Diseases.
04:10 – 04:30
Evaluating osteonecrosis using AI-based technology.
04:10 – 04:30
04:30 – 05:00
Q&A – closing remark
06:00 – 08:30
Dinner
06:00 – 08:30
Day 2
4. The Expanded Applications of AI in Lysosomal Disorders.
08:00
Arrival/Breakfast
08:00
09:00 – 9:20
Opportunities and challenges for deep learning in cell research: Creating virtual cells.
09:20 – 09:40
An AI-based approach to identify lysosomal cystine export regulating mTORC signaling
09:20 – 09:40
09:40 - 10:00
Open-Labeled Clinical Trial Using a Second-Generation Artificial-Intelligence-Based Therapeutic Regimen in Patients with Gaucher Disease Treated with Enzyme Replacement Therapy.
10:00 - 10:20
Assessment of bone involvement using the applications of Machine Learning.
10:00 - 10:20
10:20 - 10:50
Q&A
10:50 - 11:00
Coffee break
10:50 - 11:00
5. Current issues in Gene Therapies for Lysosomal Disorders.
11:00 – 11:20
Lentivirus and AAV vectors to develop Gene therapy in MPS IV.
11:00 – 11:20
11:20 – 11:40
AAV-mediated gene therapy for Sialidosis.
11:40 – 12:00
Developing a gene transfer therapy for neuronopathic disorders (MPS3c).
11:40 – 12:00
12:00 – 12:20
Patient preferences for Gene Therapy or Future of the funding and reimbursement of Gene Therapy for adult-onset LDs
12:00 - 12:20
Q&A
12:00 - 12:20
12:30
Lunch
01:30 - 05:00
GD & Bone clinic
By invitation only
01:30 - 05:00