Frontiers of Engineering Reports on Leading-Edge Engineering from the 2008 Symposium (2009) / Chapter Skim
Currently Skimming:
Polymer Technology for Gene Therapy--Daniel W. Pack
Polymer Technology for Gene Therapy--Daniel W. Pack
Pages 25-32
The Chapter Skim interface presents what we've algorithmically identified as the most significant single chunk of text within every page in the chapter.
Select key terms on the right to highlight them within pages of the chapter.
From page 25... ...
It is not difficult to envision treating monogenic diseases, such as hemophilia, muscular dystrophy, and cystic fibrosis, by replacing errant genes within the affected cells. Gene therapies are also being developed, however, for treating cardiovascular, neurological, and infectious diseases, wound healing, and cancer, by delivering genes to augment naturally occurring proteins, to alter the expression of existing genes, or to produce cytotoxic proteins or prodrug-activating enzymes.
|
From page 26... ...
In addition, at least 2 of the 11 children in the CavazzanaCalvo γc-SCID trial developed leukemia as a result of retroviral insertion of the therapeutic sequence in or near a gene associated with childhood leukemias. Thus a key limitation to the development of human gene therapy remains the lack of safe and efficient methods of gene delivery (Verma and Somia, 1997)
|
From page 27... ...
The typical endpoint of this journey is the lysosome, an acidic vesicle filled with degradative enzymes including nucleases. It is critical, therefore, that DNA and the vector escape these compartments into the cytoplasm.
|
From page 28... ...
Results, therefore, have been mixed, with few materials providing highly efficient gene delivery. A current focus in the field, therefore, is developing a new understanding of intracellular processing and polymer structure-activity relationships.
|
From page 29... ...
POLYMER TECHNOLOGY FOR GENE THERAPY 29 FIGURE 2 Structures of representative gene-delivery polymers. Pack Figure 2 R01394 bitmapped fixed image
|
From page 30... ...
. Subsequent investigation of the mechanisms leading to this unexpected enhancement revealed that PEI acetylation also decreases polymer-DNA binding strength, resulting in enhanced "unpackaging" of polyplexes within target cells.
|
From page 31... ...
2008. A combinatorial polymer library approach yields insight into nonviral gene delivery.
|
Key Terms
This material may be derived from roughly machine-read images, and so is provided only to facilitate research.
More
information on Chapter Skim is available.