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概览

疾病领域	数量

血液及淋巴系统疾病	1

排名前五的药物类型	数量

腺相关病毒基因治疗	1

排名前五的靶点	数量

factor IX(凝血因子IX)	1

关联

1

项与 Somatix Therapy Corp. 相关的药物

AAV-mediated gene therapy(Somatix Therapy)

靶点

factor IX

作用机制

凝血因子IX基因转移

在研机构

Somatix Therapy Corp.

原研机构

Somatix Therapy Corp.

在研适应症

血友病B

非在研适应症-

最高研发阶段临床前

首次获批国家/地区-

首次获批日期-

100 项与 Somatix Therapy Corp. 相关的临床结果

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0 项与 Somatix Therapy Corp. 相关的专利（医药）

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15

项与 Somatix Therapy Corp. 相关的文献（医药）

1997-08-10·Human Gene Therapy2区 · 医学

Retroviral Particles Produced from a Stable Human-Derived Packaging Cell Line Transduce Target Cells with Very High Efficiencies

2区 · 医学

Article

作者: Danos, Olivier ; Gross, Paul R. ; Spratt, S. Kaye ; Witt, Rochelle M. ; Davis, Jennifer L. ; Cohen, Lawrence K. ; Hokanson, Craig A. ; Jungles, Steven

The goal of this work was to determine whether a stable 293 amphotropic packaging line, which we have designated 293-SPA, is useful for the production of high-titer stable virus by comparison to the murine psiCRIP line. Here, we report our unexpected findings that particles derived from the 293-SPA line transduce target cells (both NIH-3T3 cells and primary melanoma cells) with greatly enhanced efficiencies (at least 10-fold) compared to particles derived from the psiCRIP packaging line. We show that the presence of a transferable inhibitor in the psiCRIP line at least partially accounts for this dramatic difference in transduction efficiency. This work has important implications for improving the efficiency of retrovirus-mediated gene transfer in general as well as in the design of new packaging cell lines.

1996-09-01·Experimental hematology4区 · 医学

High-resolution cell cycle analysis of defined phenotypic subsets within primitive human hematopoietic cell populations.

4区 · 医学

Article

作者: Jordan, C T ; Yamasaki, G ; Minamoto, D

We have developed a novel protocol for analysis of cell cycle status within specific subsets of primitive human hematopoietic cells. The technique, referred to as SID (surface, intracellular, and DNA) analysis, allows for the simultaneous characterization of cell surface and intracellular antigens, as well as quantitation of DNA content. To evaluate the technique, early human hematopoietic cells were examined using surface staining for the CD34 and CD38 antigens to identify primitive cells. Relative cell cycle status within defined phenotypic subsets (CD34+ and CD34+/CD38-) was determined by simultaneous two-parameter analysis using DNA content vs. antibody staining for the Ki-67 antigen. Ki-67 is not expressed in quiescent cells, but is quickly up-regulated as cells are induced to cycle. Consequently, expression of Ki-67, in combination with DNA content can be used to delineate all phases of the cell cycle (G0, G1, S, and GZ/M). We demonstrate that cycle induction of CD34+ cells, using IL-3, IL-6, and stem cell factor (SCF), does not correlate with activation of the CD34+CD38- subpopulation during ex vivo culture. Rather, CD34+/CD38- cells are much more refractory to cycle activation, requiring at least 72 hours to show significant levels of induction. In addition, primitive cells derived from bone marrow (BM) vs. mobilized peripheral blood (PB) show differing degrees of responsiveness to conventional ex vivo culture conditions. Finally, the effect of IL-3, IL-6, SCF, and Flt3 ligand (FL) on cycle induction was examined. It was observed that IL-3 synergized strongly with IL6+SCF to activate quiescent CD34+/CD38- cells. Moreover, when FL was combined with IL-3+IL-6+SCF, there was a small but reproducible increase in activation of CD34+/CD38- cells from G0 to G1. These data suggest that ex vivo behavior of primitive human stem cell populations is amenable to comprehensive flow cytometric analysis, and that such studies can provide detailed information on the biological response of stem cells to ex vivo culture and manipulation.

1996-08-15·Blood1区 · 医学

Transduction of primitive human hematopoietic cells with recombinant adenovirus vectors

1区 · 医学

Article

作者: Neering, SJ ; Spratt, SK ; Hardy, SF ; Jordan, CT ; Minamoto, D

We have examined the ability of recombinant adenoviral vectors to transduce human hematopoietic cells. Our findings indicate that adenovirus readily infects a large proportion of CD34+ cells. Using adenovirus vectors that transduce either a lacZ or an alkaline phosphatase reporter gene, we observed up to 45% of total CD34+ cells infected. Upon more detailed analysis, we observed comparable levels of transduction for CD34+/CD38- cells and for CD34+ cells in G(zero) phase of the cell cycle. Importantly, exposure to adenovirus resulted in negligible levels of toxicity as assayed by propidium iodide staining and colony-forming ability. Using adenovirus vectors, we also describe a model system for regulated gene expression in early hematopoietic tissues. CD34+ cells were simultaneously infected with two viruses, one carrying a TetR/VP16 transactivator (tTA) and the second carrying a tTA- dependent lacZ reporter gene. Using this approach, beta-gal expression was only observed upon coinfection with the transactivator vector. In addition, as shown previously (Gossen and Bujard, Proc Natl Acad Sci USA 89:5547, 1992), tetracycline was able to inhibit tTA mediated induction, thereby providing an effective means to regulate expression of the reporter gene. We conclude that recombinant adenovirus is an effective vehicle for transiently expressing genes in primitive human hematopoietic cells.

100 项与 Somatix Therapy Corp. 相关的药物交易

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100 项与 Somatix Therapy Corp. 相关的转化医学

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