Novel retrovirus associated with myelodysplastic syndromes and uses thereof

Abstract

The present invention relates first, to the identification of a novel retrovirus and the novel nucleotide sequences encoding a retroviral polymerase gene (POL nucleotides) associated with the existence of MDS and MDS associated disorders. The present invention further relates to methods for using the MDS associated retroviral nucleotides for the detection of MDS and MDS associated disorders in patient samples. The present invention also relates to methods for using and targeting the MDS associated retroviral POL nucleotides in gene therapy protocols for the treatment of MDS and MDS associated disorders in patients in need of such treatment. The present invention further relates to diagnostic protocols and kits for the detection of MDS and MDS associated disorders in tissue samples.

Description

[0001] This application is a continuation of U.S. Ser. No. 10 / 433,244, filed May 28, 2003, which is a national stage application of International Application PCT / US01 / 44263, filed Nov. 27, 2001, which claims priority to U.S. Provisional Patent Application Ser. No. 60 / 253,591, filed Nov. 28, 2000, the entire contents of which are hereby incorporated by reference.INTRODUCTION [0002] The present invention relates first, to the identification of a novel retrovirus and associated reverse transcriptase activity associated with the existence of myelodysplastic syndromes (MDS). The present invention further relates to methods for using the novel retrovirus and its associated reverse transcriptase activity and retroviral nucleotides for the detection of MDS in patient samples. The present invention further relates to nucleotide sequences encoding the MDS associated retrovirus, in particular novel nucleotide sequences encoding a retroviral polymerase gene associated with MDS and acute leukemi...

AI Technical Summary

Benefits of technology

The novel retrovirus-based approach enables effective detection and potential treatment of MDS, addressing the cytopenias and disease progression by targeting the retroviral activity, offering a new avenue for managing MDS and associated leukemias.

Problems solved by technology

Thus, even though the bone marrow is working overtime to produce more and more blood cells, they are “ineffective” since they begin to die prematurely.

Due to the complexity of MDS, no single therapeutic approach appears to have made a significant impact on survival of patients with MDS.

The problem is that administered as single agents, granulocyte-macrophage colony-stimulating factor (GM-CSF) or G-CSF rarely improves the anemia and the thrombocytopenia so commonly the pathognomonic features of MDS.

However, only a proportion of patients respond, the response is usually temporary, and there is some concern related to an incidence of accelerated transformation.

Short duration of remission marked by a relentless return of MDS cells in most patients, treatment-related complications or mortality, frequent encounters with drug-resistant clones, and the morbidity caused by the appearance of unexpected and unusual opportunistic infections reflecting the enormously compromised state of the immune system in these patients make the intensive chemotherapy option less desirable.

In summary, save for allogeneic transplantation, MDS is a universally fatal illness, and no single approach has either altered the natural history of the disease or improved survival.

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