Crystal's "Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis" 1994

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===Abstract===
===Abstract===
-
*Just put an asterisk as the first character to make a bullet point.
+
*They administered a recombinant adenovirus vector (AdCFTR) containing the normal human CFTR cDNA into the nasal and bronchial epithelium of 4 individuals with CF.
-
**Put two asterices to make a sub point.
+
*They found the vector expresses the CFTR cDNA in the respiratory epithelium in vivo.
-
*Put three single quote marks around text you want to be '''bold''', perhaps for '''new terminology'''.
+
*At 2x10<sup>9</sup> pfu there was no recombination, complementation, shedding of the vector, or rise in antibody titres. Although, there was a transient and systemic pulmonary syndrome observed (possibly mediated by IL-6)
-
*Put two single quotes around ''things that should be italicized''.
+
*They saw no long term effects
===Introduction===
===Introduction===
 +
*CF is a common lethal hereditary disorder caused by a mutation on CFTR on chromosome 7
 +
*The disorder is characterized by airway and gastrointestinal disease, the lung manifestations dominate
 +
*The pathogenesis is clearly linked to the lack of CFTR in the respiratory epithelia
 +
*Symptoms in first decade:
 +
**Thick mucus, colonization with infectious bacteria, and chronic airway inflammation
 +
*One approach to prevent respiratory manifestations of CFTR is gene therapy (talked about in abstract)
 +
*Gene therapy must be carried out ''in vivo'', cannot be done ''ex vivo''
 +
 +
====I also decided to add in the notes I took in class just for completeness====
 +
*One of the four human gene therapy trials approved and initiated at the same time
 +
*Based on the results of this trial, the others were halted
 +
*Used CF patients who were in remarkably good health
 +
*Did multiple dosing to find effecting concentration
 +
 +
====Results in the human study:====
 +
*Treatment evoked an immune response
 +
*Inflammation accompanied the immune response
 +
*Results are short lived-- at most 6 weeks
 +
*Because of the immune response, will not be able to do multiple dosing
 +
*Very inefficient transfer of gene of interest-- will do nothing to correct the defect
 +
*"Correction of the CF phenotype of the airway epithelium might be achieved with this strategy"
 +
*"To maintain chronic expression, adenovirus vectors will probably have to be administered repeatedly"
 +
**This is not possible with an immune response

Current revision as of 19:30, 7 March 2010

Contents

[edit] Abstract

  • They administered a recombinant adenovirus vector (AdCFTR) containing the normal human CFTR cDNA into the nasal and bronchial epithelium of 4 individuals with CF.
  • They found the vector expresses the CFTR cDNA in the respiratory epithelium in vivo.
  • At 2x109 pfu there was no recombination, complementation, shedding of the vector, or rise in antibody titres. Although, there was a transient and systemic pulmonary syndrome observed (possibly mediated by IL-6)
  • They saw no long term effects

[edit] Introduction

  • CF is a common lethal hereditary disorder caused by a mutation on CFTR on chromosome 7
  • The disorder is characterized by airway and gastrointestinal disease, the lung manifestations dominate
  • The pathogenesis is clearly linked to the lack of CFTR in the respiratory epithelia
  • Symptoms in first decade:
    • Thick mucus, colonization with infectious bacteria, and chronic airway inflammation
  • One approach to prevent respiratory manifestations of CFTR is gene therapy (talked about in abstract)
  • Gene therapy must be carried out in vivo, cannot be done ex vivo

[edit] I also decided to add in the notes I took in class just for completeness

  • One of the four human gene therapy trials approved and initiated at the same time
  • Based on the results of this trial, the others were halted
  • Used CF patients who were in remarkably good health
  • Did multiple dosing to find effecting concentration

[edit] Results in the human study:

  • Treatment evoked an immune response
  • Inflammation accompanied the immune response
  • Results are short lived-- at most 6 weeks
  • Because of the immune response, will not be able to do multiple dosing
  • Very inefficient transfer of gene of interest-- will do nothing to correct the defect
  • "Correction of the CF phenotype of the airway epithelium might be achieved with this strategy"
  • "To maintain chronic expression, adenovirus vectors will probably have to be administered repeatedly"
    • This is not possible with an immune response
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