NATIONAL TAIWAN UNIVERSITY HOSPITAL
CD19-targeted CAR T-cell therapeutics for hematologic malignancies
The Application of Electrospun Biomaterial-PBG on the Corneal Nerve Regeneration
Until now, there have been no effective therapies for neurological disorders. As for peripheral nerve diseases, although not causing death directly, severely impact patients' health and quality of life. Our team has developed a biomedical material suitable for neural repair called the “Electrospun Biomedical Scaffold - PBG.” PBG exhibits good plasticity, biocompatibility, and degradability. It can sustained-release the excitatory neurotransmitter “glutamate” which promotes neural growth and induces the differentiation of iPSCs into retinal ganglion cells. Furthermore, it has shown the ability to repair damaged trigeminal nerves in in vitro experiments. In animal transplantation experiments, the PBG scaffold was demonstrated to directly promote the regeneration of damaged corneal nerves. The “Electrospun Biomedical Scaffold - PBG” holds great potential to become an “Electrospun Scaffold Artificial Cornea” that aids in the repair of damaged corneal nerves.
Gene therapy for AADC deficiency
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic neurological disorder caused by autosomal recessive inherited pathogenic mutants of the DDC gene, which prevents the conversion of L-3,4-dihydroxyphenylalanine (L-DOPA) into dopamine (DA) that leads to decreased availability of serotonin and dopamine in the presynaptic and synaptic cleft, as well as a deficiency of catecholamines. AADC deficiency results in movement disorders including hypokinesia, dystonia, and oculogyric crisis, along with behavioral problems, autonomic dysfunction, and developmental delay. The rationale for intraputaminal AADC gene therapy is to deliver a functional copy of the human DDC gene (hAADC) with a recombinant adeno-associated virus type 2 (AAV2) vector directly to striatal regions impacted by the disease, which subsequently leads to increased conversion of levodopa to DA in targeted striatal neuronal cell bodies. Patients received AAV2-hAADC infused bilaterally into the putamen. The procedure and treatment were well tolerated. An increase in dopamine production could be demonstrated by positron emission tomography and neurotransmitter analysis.