PubMed Health⌕ Search

PubMed · 9398067

Gene therapy.

Abstract

In recent years, there have been a number of technological breakthroughs that have allowed for clinical trials in gene therapy to be initiated. In combination with the genome initiative, the potential for new therapeutics is limitless. Although an enormous amount of information has been obtained in a relatively short period of time, gene therapy is not yet ready for wide-scale practice. Some of the successes and obstacles that remain are summarized in this report.

Explore related subjects

Keep this discovery

Explore connections, maps & timelines

BibTeXRIS

M A Kay, D Liu, P M Hoogerbrugge. 1997-11-25. Gene therapy.. https://doi.org/10.1073/pnas.94.24.12744

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

KEEP EXPLORING

Related citations

MRI findings in Shwachman diamond syndrome.

Shwachman Diamond Syndrome (SDS) is a rare congenital disorder characterized by pancreatic insufficiency, bone marrow dysfunction, and skeletal changes. Because of the heterogeneous clinical presentation and the limits of laboratory tests that assess pancreatic insufficiency, the diagnosis of SDS can be challenging. Pancreatic lipomatosis, a typical feature of this syndrome, is also difficult to assess by direct tissue sampling. In these circumstances, magnetic resonance imaging (MRI) provides a readily available, noninvasive tool to evaluate the pancreatic fat content. We report a case of a 12-month-old male in which abdominal MRI was used to confirm the clinical diagnosis of SDS.

Bone Marrow Diseases↗

Nuclear medicine imaging and therapy of neuroendocrine tumours.

Radiolabelled peptides are used for specific targeting of receptors (over-)expressed by tumour cells. Dependent on the kind of labelling and the radionuclide used, these compounds may be utilised for imaging or for therapy. A concise overview is provided on basic principles of designing and developing radiopeptides for these applications. Furthermore, clinical application of these compounds for imaging and therapy is described. Advantages of the method compared to other techniques (such as the use of radiolabelled antibodies or antibody fragments) are discussed as well as pitfalls and limitations.

Bone Marrow Diseases↗