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 micro-organ is transduced in vitro with a helper-dependent adenoviral (HdAd) vector or with an adeno-associated viral (AAV) vector compris

1. A long-lasting therapeutic formulation for implanting into an immunocompetent human subject comprising a genetically modified dermal micro-organ, wherein said micro-organ is transduced in vitro with a helper-dependent adenoviral (HdAd) vector or with an adeno-associated viral (AAV) vector comprising a nucleic acid sequence encoding a therapeutic erythropoietin polypeptide operably linked to one or more regulatory sequences, wherein said nucleic acid sequence is greater than 95% homologous to SEQ ID No: 1, wherein following said transduction in vitro expression of said therapeutic polypeptide is determined, 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 therapeutic erythropoietin in the subject's serum compared with pre-implantation basal levels; 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. 3. The formulation of claim 1 wherein said micro-organ is implanted subcutaneously. 4. The formulation of claim 1, wherein said micro-organ is implanted intradermally. 5. The formulation of claim 1, wherein said one or more regulatory sequences comprise a CAG promoter. 6. The formulation of claim 1, wherein said micro-organ is transduced in vitro with an HdAd vector. 7. The formulation of claim 1, wherein said micro-organ is transduced in vitro with an AAV vector. 8. The formulation of claim 5, wherein the in vitro expression of said therapeutic erythropoietin polypeptide is increased under the control of a CAG promoter compared to a CMV promoter.