Gene therapy: trials and tribulations

N Somia, IM Verma - Nature Reviews Genetics, 2000 - nature.com
… a gene therapy Phase I clinical trial has overshadowed some recent successes in gene therapy
… The next phase of gene therapy will be focused on targeted and regulated expression of …

Cystic fibrosis gene therapy trials and tribulations

PL Zeitlin - Molecular Therapy, 2000 - cell.com
gene expression in other gene therapy trials. Are there comparable changes in CFTR function
from pharmacological trials … What should we expect from sufficient CFTR gene expression …

Trials and tribulations

S Gilbert - The Hastings Center Report, 2008 - JSTOR
… 1999 during a gene therapy trial at the Universi ty of Pennsylvania. Gelsinger's death was
the first to be definitively linked to a gene therapy trial. Would Mohr's be another? The trial was …

[引用][C] Trials and tribulations: the cost of developing gene therapies

MT Lotze - The FASEB journal, 1991 - Wiley Online Library
gene therapies using viral vectors to insert genes into human cells. In fact gene therapy, long
… months before clinical centers around the country will begin their own trials of gene transfer …

Gene therapy for hemophilia: clinical trials and technical tribulations

NO Viiala, SR Larsen, JEJ Rasko - Seminars in thrombosis and …, 2009 - thieme-connect.com
… have been reports of more significant complications in other gene therapy applications. In
an unrelated gene therapy trial using an adenoviral vector to treat ornithine transcarbamylase …

Cystic fibrosis gene therapy trials and tribulations

NJ Caplen - Trends in Molecular Medicine, 2001 - cell.com
Cystic fibrosis (CF) has long been seen as a candidate for treatment by gene therapy.
Reasons for this include: the genetics of CF are simple; it is an autosomal recessive disorder …

Trials and Tribulations in Developing Clinical Trials of Gene Therapy: E1A for Breast or Ovarian Cancer

NT Ueno, JK Wolf, DM Gershenson… - Gene Therapy for …, 2007 - Springer
… In our phase I trial of E1A gene therapy, delivery of the E1A gene into either the thoracic or
the peritoneal cavity by means of DC-Chol cationic liposomes led to successful transfection of …

Trials and Tribulations: An Industry Perspective on Conducting Registrational Trials in Alpha-1 Antitrypsin Deficiency

MS Forshag - Annals of the American Thoracic Society, 2016 - atsjournals.org
… of genetic origin that carries the additional challenges of variable penetration and slow
disease progression. Registrational trials of augmentation therapytherapy in many countries. …

Angiogenesis Therapies for Coronary Artery Disease: Trials and Tribulations

M Simons - Cardiac Drug Development Guide, 2003 - Springer
… for VEGF as a therapeutic agent. Embryonic lethality in VEGF gene deletions occurs for a …
administration to local protein delivery or gene therapy approaches. Finally, recent reports …

Gene therapy: promises and problems

A Pfeifer, IM Verma - Annual review of genomics and human …, 2001 - annualreviews.org
… In this review, we focus on viralvectors and highlight some examples of their use in clinical
trials. Acomplete, constantly updated list of human gene therapy trials in the UnitedStates is …