PubMed Health⌕ Search
Home/Archive/PubMed

PubMed · 11449669

Gene therapy.

E V Badiavas·V Falanga
View original source ↗Export metadata ↓

Abstract

With recent advances in molecular biology, the ability to transfer genes to patients is becoming a reality. Ongoing clinical trials using gene transfer techniques have illustrated the potential and pitfalls of this new therapeutic modality for the treatment of a wide variety of disorders. While these techniques are not currently a part of routine clinical practice, it is only a matter of time until some form of gene therapy is approved for general use in the clinic. This review highlights some of the basic methods used in current gene therapy protocols. The objective of this review is to familiarize practitioners with these concepts so they can more effectively follow the progress of this emerging technology and better inform their patients.

Explore related subjects

Gene Transfer TechniquesGenetic TherapyGenetic VectorsHumansJournal ArticleReview

Keep this discovery

Explore connections, maps & timelines

BibTeXRIS

E V Badiavas, V Falanga. 2001. Gene therapy.. https://doi.org/10.1111/j.1346-8138.2001.tb00116.x

Cite the original work for its findings. Save a collection to share your selection of sources.

KEEP EXPLORING

Related citations

PubMed2003

Pluronic-grafted poly-(L)-lysine as a new synthetic gene carrier.

Genes are attractive candidates as therapeutic agents, and the development of safe and effective gene carriers is essential for the success of human gene therapy. To develop a gene delivery vector that shows low cytotoxicity and high efficiency, we synthesized poly-L-lysine-g-pluronic by conjugating poly-L-lysine (PLL) to pluronic, which is partially functionalized with para-nitrophenyl carbonate groups, and evaluated for its efficiency as a possible nonviral gene carrier candidate. Structural analysis of synthesized polymer was performed by using 1H-NMR. Gel retardation assay, zeta potential and size measurement confirmed that the new gene carrier made a compact complex with plasmid DNA. pCMV-beta-gal was used as a reporter gene, and the in vitro transfection efficiency was measured in HeLa cells by using the o-nitrophenyl-beta-D-galactopyranoside assay. The highest transfection efficiency among those tested was achieved at the 1:1 weight ratio of polymer:DNA, and a 3-fold increase in transfection efficiency was achieved by treatment of a lysosomotropic agent, chloroquine. Compared with unmodified PLL, PLL-g-pluronic showed about 2-fold increase in transfection efficiency with similar cytotoxicity specifically at the 1:1 weight ratio of polymer:DNA.

Gene Transfer Techniques↗
PubMed2003

Expression of the gamma-zein protein of maize in seeds of transgenic barley: effects on grain composition and properties.

A cDNA clone encoding the gamma-zein protein of maize was expressed in developing grain of barley using the starchy endosperm cell-specific promoter from the wheat Glu-1D-1 (HMW subunit 1Dx5) gene. Seven transgenic lines were recovered from 226 bombarded immature embryos, of which two were sterile and four tetraploid, while five were shown to express the gamma-zein protein based on western blotting. Southern blot analysis showed the presence of between about three and twelve transgene insertions. Detailed comparative studies of five null and five homozygous transformed sub-lines from transgenic line A showed that gamma-zein accounted for over 4% of the total prolamin fraction, corresponding to about 1.9% of the total grain N. Comparison of the proteins present in the gel protein fraction demonstrated that the gamma-zein was incorporated into polymers, as in maize. However, there was no effect on grain hardness measured using the Perten Single Kernel Characterisation System or on the vitreousness measured by visual inspection. This contrasts with the situation in maize where a clear association with vitreousness has been reported.

Gene Transfer Techniques↗