University of Pennsylvania

Fondazione Telethon

Markus Hildinger

Alberto Auricchio

James M Wilson

Enrico Maria Surace

The present invention concerns the use of compounds that interfere with the hedgehog signaling pathway for the manufacture of a medicament for preventing, inhibiting, and/or reversing ocular diseases related with ocular neovascularization. Particularly, the above-mentioned diseases are (wet) age-related macular degeneration, (proliferative) diabetic retinopathy, neovascular glaucoma, retinal vein occlusion, or retinopathy of prematurity (ROP).

Use of compounds that interfere with the hedgehog signaling pathway for the manufacture of a medicament for preventing, inhibiting, and/or reversing ocular diseases related with ocular neovascularization

A method for isolating, from a mixture, a virus having a surface protein with a binding site for sialic acid is provided. The method involves contacting the mixture with mucin which has been linked to a solid support and washing the solid support to remove material from the mixture is non-specifically bound to the mucin-linked support. Thereafter, the specifically bound virus (e.g., AAV4 or AAV5) may be removed in a further washing step utilizing a concentrated slat or solution with low pH. Also described are pharmaceutical kits containing solid supports linked to mucin for use in isolating virus having a surface protein with a binding site for sialic acid, or detecting the presence of the virus in a biological sample.

Method for purification of viral vectors having proteins which bind sialic acid

A pseudotyped rAAV is described, which contains sequences derived from AAV5. A method for producing rAAV pseudotyped with AAV5 capsid is described in which the rep proteins of the serotype or any cross-reactive serotype of the AAV ITRs are utilized. A similar method may be provided to generate a pseudotyped rAAV in which minigenes carrying AAV5 ITRs are packaged in capsids of a heterologous AAV serotype. Also provided are pharmaceutical compositions containing the pseudotyped rAAV and methods of using them for gene delivery.

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Boston, MA, United States of America

Markus Hildinger