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US 2016/0101174 A1Published application
Lentiviral vectors with tropism to motor neurons comprising an antibody that binds to a pre-synaptic terminal receptor on the neuromuscular junction and a fusogenic protein
OwnerNicholas D. MAZARAKIS, Ioanna ELEFTHERIADOU +1inventors
InventorsNicholas D. MAZARAKIS, Ioanna ELEFTHERIADOU, Antonio TRABALZA
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Abstract
The present invention relates to vectors useful in the treatment of disease through targeted delivery. In particular, the present invention relates to vectors useful in the treatment of disease associated with neuronal degeneration, such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA).
Claims21
A lentiviral vector comprising (i) an antibody that binds to a pre-synaptic terminal receptor on the neuromuscular junction (NMJ);
- and (ii) a fusogenic molecule.
(canceled)
Dependencies are inferred from the claim text.