초록 ▼

The present invention is directed to long-lasting therapeutic formulations and their methods of use wherein the formulation comprises a genetically modified micro-organ that comprises a vector which comprises a nucleic acid sequence operably linked to one or more regulatory sequences, wherein the nu

The present invention is directed to long-lasting therapeutic formulations and their methods of use wherein the formulation comprises a genetically modified micro-organ that comprises a vector which comprises a nucleic acid sequence operably linked to one or more regulatory sequences, wherein the nucleic acid sequence encodes a therapeutic polypeptide, such as erythropoietin or interferon alpha.

대표청구항 ▼

1. A long-lasting therapeutic formulation for implanting into an immunocompetent human subject comprising: a genetically modified dermal micro-organ, wherein said dermal micro-organ consists essentially of dermal tissue and lacks a complete epidermal layer, wherein said dermal micro-organ is transdu

1. A long-lasting therapeutic formulation for implanting into an immunocompetent human subject comprising: a genetically modified dermal micro-organ, wherein said dermal micro-organ consists essentially of dermal tissue and lacks a complete epidermal layer, wherein said dermal micro-organ is transduced in vitro with a helper-dependent adenoviral vector comprising the nucleic acid sequence set forth in SEQ ID NO: 2, wherein said nucleic acid sequence encodes for interferon and is operably linked to one or more regulatory sequences, wherein said one or more regulatory sequences comprise a CAG promoter, wherein following said transduction the genetically modified dermal micro-organ is maintained ex vivo prior to implantation in order to reduce the immunogenicity of said formulation and wherein implantation of said long-lasting formulation in said immunocompetent human subject provides a beneficial effect selected from the group consisting of:a. an increase in expression levels of said interferon in the serum compared with pre-implantation basal levels, said increase persisting for greater than one month; andb. an alleviation of a symptom of a disease or disorder in said subject, wherein said alleviation persists for greater than one month. 2. The formulation of claim 1, wherein said regulatory sequence comprises a SV40 polyadenylation sequence.