Abstract
THE treatment of inherited enzyme deficiencies in man can be approached in at least two ways: correction of the malfunctioning gene normally responsible for the transcription of the missing enzyme, or exogenous replacement of the missing enzyme1. In both approaches enzymes, DNA or RNA fragments or drugs designed to replace or correct those that are missing or malfunctioning, should, after administration, reach and act in the cells where the enzyme function is important. In reaching the site of action, however, such substances could provoke immunological reactions, be incompatible with blood or vulnerable to blood enzymes, be taken up by the reticuloendothelial system or non-diseased tissues, or even be unable to reach or penetrate target tissues.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 51 print issues and online access
$199.00 per year
only $3.90 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Similar content being viewed by others
References
Strauss, B. S., in Lipid Storage Diseases (edit. by Bernsohn, J., and Grossman, H. J.), 291 (Academic, New York, 1971).
Gregoriadis, G., Leathwood, P. D., and Ryman, B. E., FEBS Lett., 14, 95 (1971).
Gregoriadis, G., and Ryman, B. E., Biochem. J., 124, 58p (1971).
Gregoriadis, G., and Ryman, B. E., Eur. J. Biochem., 24, 485 (1972).
Gregoriadis, G., and Ryman, B. E., Biochem. J., 129, 123 (1972).
Magee, W. E., and Miller, O. V., Nature, 235, 339 (1972).
Bangham, A. D., Progr. Biophys. Mol. Biol., 18, 19 (1968).
Sessa, G., and Weissman, G., J. Lipid Res., 9, 310 (1968).
Hers, H. G., and Van Hoof, F., in Progress in Liver Diseases (edit. by Popper, H., and Schaffner, F.), 185 (Heinemann, London, 1970).
Brady, R. O., in Lipid Storage Diseases (edit. by Bernsohn, J., and Grossman, H. J.), 275 (Academic, New York, 1971).
Cohn, Z. A., and Ehrenreich, B. A., J. Exp. Med., 129, 201 (1969).
Nyberg, W. E., and Dingle, J. T., Exp. Cell Res., 63, 43 (1970).
Cohn, Z. A., and Benson, B., J. Exp. Med., 121, 153 (1965).
Lowry, O. H., Rosebrough, N. J., Far, A. L., and Randall, R. J., J. Biol. Chem., 193, 265 (1951).
Patterson, M. S., and Greene, R. C., Anal. Chem., 37, 854 (1965).
Raivio, K. O., and Seegmiller, E., Ann. Rev. Biochem., 41, 543 (1972).
Ehrenreich, B. A., and Cohn, Z. A., J. Exp. Med., 126, 941 (1967).
Smith, I., in Chromatographic and Electrophoretic Techniques (edit. by Smith, I.), 1, 246 (Heinemann, London, 1961).
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
GREGORIADIS, G., BUCKLAND, R. Enzyme-containing Liposomes alleviate a Model for Storage Disease. Nature 244, 170–172 (1973). https://doi.org/10.1038/244170a0
Received:
Revised:
Issue Date:
DOI: https://doi.org/10.1038/244170a0
This article is cited by
Comments
By submitting a comment you agree to abide by our Terms and Community Guidelines. If you find something abusive or that does not comply with our terms or guidelines please flag it as inappropriate.
