C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/113 (2010.01) C12N 5/071 (2010.01) C12N 5/0735 (2010.01) A61K 31/4015 (2006.01) A61K 31/69 (2006.01) A61K 31/706 (2006.01) A61K 31/7068 (2006.01) A61K 38/05 (2006.01) A61K 38/06 (2006.01) A61K 38/07 (2006.01) A61P 35/00 (2006.01) C12Q 1/00 (2006.01) C12Q 1/68 (2006.01)
Patent
CA 2690725
The present invention discloses nucleic acids, proteins, and antibodies for SALL4 (including isoforms SALL4A, SALL4B, and SALL4C), a zinc finger transcriptional factor. Further, methods are disclosed which demonstrate that constitutive expression of SALL4 increases leukemogenic potential in cells of model animal systems. Moreover, constitutive expression of select isoforms (e.g., SALL4B) in transgenic mice demonstrate that these animals develop myelodysplastic syndrome (MDS)-like signs and symptoms, including subsequent acute myeloid leukemia (AML), which is transplantable. The disclosure also provides methods for identifying and purifying embryonic stem cells, adult stem cells, cancer stem cells, including leukemia stem cells, methods for identifying substances which bind to and/or modulate SALL4, methods for diagnosing MDS in a subject, and methods of treating a subject presenting MDS, AML and other forms of leukemia.
La présente invention concerne des acides nucléiques, des protéines et des anticorps pour SALL4 (y compris les isoformes SALL4A, SALL4B et SALL4C), un facteur de transcription à doigt de zinc. L'invention concerne également des méthodes démontrant que l'expression constitutive de SALL4 augmente le potentiel leucémogène dans les cellules de systèmes modèles animaux. Par ailleurs, l'expression constitutive d'isoformes sélectionnées (par ex., SALL4B) dans des souris transgéniques démontre que ces animaux développent des signes et des symptômes de type syndrome myélodysplasique (SMD), et notamment une leucémie myéloïde aiguë (LMA) subséquente, qui est transplantable. L'invention se rapporte en outre à des méthodes d'identification et de purification de cellules souches embryonnaires, de cellules souches adultes et de cellules souches cancéreuses, telles que des cellules souches leucémiques, ainsi que des méthodes de diagnostic d'un SMD chez un sujet et des méthodes de traitement d'un sujet présentant un SMD, une LMA et d'autres formes de leucémie.
Mbm Intellectual Property Law Llp
Nevada Cancer Institute
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