Results 11 to 20 of about 1,182,904 (110)

Gene Therapy

open access: yesHematology/Oncology Clinics of North America, 2017
Gene therapy refers to a rapidly growing field of medicine in which genes are introduced into the body to treat or prevent diseases. Although a variety of methods can be used to deliver the genetic materials into the target cells and tissues, modified viral vectors represent one of the more common delivery routes because of its transduction efficiency ...
Barb, Thorne   +3 more
  +18 more sources

Gene therapy [PDF]

open access: yesProceedings of the National Academy of Sciences, 1997
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 ...
M A, Kay, D, Liu, P M, Hoogerbrugge
openaire   +2 more sources

Gene therapy. [PDF]

open access: yesGut, 1982
Gene therapy is not yet possible, but may become feasible soon, particularly for well understood gene defects. Although treatment of a patient raises no ethical problems once it can be done well, changing the genes of an early embryo is more difficult, controversial and unlikely to be required clinically.
openaire   +4 more sources

Gene therapy

open access: yesBiology of Blood and Marrow Transplantation, 1999
For the past two decades, concerted efforts have been made to treat human disease by replacing nonfunctioning genes in cells or by correcting mutations that produce disease. Successful application of these methods could lead to effective therapies for a variety of genetic and acquired diseases, many of which are not treatable today.
openaire   +2 more sources

Cochlear Gene Therapy [PDF]

open access: yesCold Spring Harbor Perspectives in Medicine, 2012
Over 450 million people worldwide suffer from hearing loss, leading to an estimated economic burden of ∼$750 billion. The past decade has seen significant advances in the understanding of the molecular mechanisms that contribute to hearing, and the environmental and genetic factors that can go awry and lead to hearing loss.
Lawrence R, Lustig, Omar, Akil
openaire   +4 more sources

Gene therapy [PDF]

open access: yesOral Diseases, 2013
Applications of gene therapy have been evaluated in virtually every oral tissue, and many of these have proved successful at least in animal models. While gene therapy will not be used routinely in the next decade, practitioners of oral medicine should be aware of the potential of this novel type of treatment that doubtless will benefit many patients ...
openaire   +2 more sources

Gene therapy for arthritis [PDF]

open access: yesExpert Opinion on Investigational Drugs, 1994
In the two years since arthritis gene therapy was last reviewed in this journal, there has been rapid progress on several fronts. Although vector development remains a slow process and long-term gene expression is not easily obtained, very encouraging preclinical data in animal models of arthritis are now emerging.
C H, Evans, P D, Robbins
openaire   +2 more sources

Is it ‘gene therapy’?

open access: yesJournal of Law and the Biosciences, 2018
What, exactly, is "gene therapy"? Crystallizing a modern definition of "gene therapy" has now become critically important on several fronts. Following the recent of passage the 21st Century Cures Act, FDA now relies on the term to give regulatory incentives to sponsors of certain ‘regenerative medicines’, including ‘gene therapies’.
Jacob S Sherkow   +2 more
openaire   +3 more sources

Gene Therapy for Blindness

open access: yesAnnual Review of Neuroscience, 2013
Sight-restoring therapy for the visually impaired and blind is a major unmet medical need. Ocular gene therapy is a rational choice for restoring vision or preventing the loss of vision because most blinding diseases originate in cellular components of the eye, a compartment that is optimally suited for the delivery of genes, and many of these diseases
Sahel José-Alain, Roska Botond
openaire   +3 more sources

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