CournoyerDScarpaMJonesSNMooreKABelmontJWCaskeyCT. Gene therapy: A new approach for the treatment of genetic disorders. Clin Pharmacol Ther1990; 47: 1–11.
2.
MarxJL. Getting to the heart of genetic disease. Science1989; 243: 315–6.
3.
CrystalRG. Alpha1-antitrypsin deficiency, emphysema, liver disease: Genetic basis and strategies for therapy. J Clin Invest1990; 85: 1343–52.
4.
HubbardRCBrantlyMLSellersSEMichellMECrystalRG. Delivery of proteins for therapeutic purposes by aerosolization: Direct augmentation of anti-neutrophil elastase defenses of the lower respiratory tract in alpha,-antitrypsin deficiency with an aerosol of alpha1-antitrypsin. Ann Intern Med1989; 111: 206–12.
5.
HussarDA. New drugs of 1991. Am Pharm1992; NS32 (4):317–50.
6.
BeutlerE. Gaucher disease: New molecular approaches to diagnosis and treatment. Science1992; 256: 794–9.
7.
Genzyme files for Ceredase in Europe. Scrip1992;(Apr 15): 17.
8.
HiltsPJ. Seeking limits to a drug monopoly. New York Times1992 May 12; Sect C:1.
9.
Cystic fibrosis. In: BerkowR, ed. Merck manual of diagnosis and therapy. Rahway, NJ: Merck Sharp & Dohme Research Laboratories, 1987: 2055–9.
SnouwaertJNBrigmanKKLatourAMMaloufNNBoucherRCSmithiesO. An animal model for cystic fibrosis made by gene targeting. Science1992; 257: 1083–8.
12.
ValeriusNHKochCHoibyN. Prevention of chronic Pseudomonas aeruginosa colonisation in cystic fibrosis by early treatment. Lancet1991; 338: 725–6.
13.
CryzSJJrSadoffJCCrossASFurerE. Safety and immunogenicity of a polyvalent Pseudomonas aeruginosa O-polysaccharide-toxin A vaccine in humans. Antibiot Chemother1989; 42: 177–83.
14.
SchaadUBLangABWedgwoodJRuedebergAQueJUFurerE. Safety and immunogenicity of Pseudomonas aeruginosa conjugate A vaccine in cystic fibrosis. Lancet1991; 338: 1236–7.
15.
Choosing and using a pancreatic enzyme supplement. Drug Ther Bull1992; 30: 37–40.
16.
HendelesLDorfAStecenkoAWeinbergerM. Treatment failure after substitution of generic pancrelipase capsules. Correlation with in vitro lipase activity. JAMA1990; 263: 2459–61.
17.
RheinsteinPH. Regulatory status of pancreatic enzyme preparations. JAMA1990; 263: 2491–2.
18.
DavisPB. Cystic fibrosis from bench to bedside. N Engl J Med1991; 325: 575–7.
19.
WaltnerWEBoucherRCGatzyJTKnowlesMR. Pharmacotherapy of airway disease in cystic fibrosis. Trends Pharmacol Sci1987; 8: 316–20.
20.
KnowlesMRChurchNLWaltnerWEYankaskasJRGilliganPKingM. A pilot study of aerosolized amiloride for the treatment of lung disease in cystic fibrosis. N Engl J Med1990; 322: 1189–94.
21.
Aerosolized amiloride plus ATP or UTP for cystic fibrosis. FDC Reports. Pink Sheet1991;(Aug 26): T&G5–6.
22.
HwangT-CZeitlinPLGruenertDCHuganirRCugginoWB. Cl− channels in CF: Lack of activation by protein kinase C and cAMP-dependent protein kinase. Science1989; 244: 1351–3.
23.
MasonSJParadisoAMBoucherRC. Regulation of transepithelial ion transport and intracellular calcium by extracellular adenosine triphosphate in human normal and cystic fibrosis airway epithelium. Br J Pharmacol1991; 103: 1649–56.
24.
KnowlesMRClarkeLLBoucherRC. Activation by extracellular nucleotides of chloride secretion in the airway epithelia of patients with cystic fibrosis. N Engl J Med1991; 325: 533–8.
25.
ShakSCaponDJHellmissRMarstersSABakerCL. Recombinant human DNase I reduces the viscosity of cystic fibrosis sputum. Proc Natl Acad Sci U S A1990; 87: 9188–92.
26.
HubbardRCMcElvaneyNGBirrerPShakSRobinsonWWJolleyC. A preliminary study of aerosolized recombinant human deoxyribonuclease I in the treatment of cystic fibrosis. N Engl J Med1992; 326: 812–5.
27.
RubinBK. Aerosolized recombinant human deoxyribonuclease I in the treatment of cystic fibrosis (letter). N Engl J Med1992; 327: 571.
28.
HubbardRCShakSCrystalRG. Aerosolized recombinant human deoxyribonuclease I in the treatment of cystic fibrosis (letter). N Engl J Med1992; 327: 571.
29.
AitkenMLBurkeWMcDonaldGShakSMontgomeryABSmithA. Recombinant human DNase inhalation in normal subjects and patients with cystic fibrosis: A phase I study. JAMA1992; 267: 1947–51.
30.
DNase on fast-pace for cystic fibrosis PLA filing: Application to FDA planned for early 1993; DNase will be biotech's “next major product,” Genentech says. FDC Reports. Pink Sheet1992;(May 11):9–10.
31.
McElvaneyNGHubbardRCBirrerPChernickMSCaplanDBFrankMM. Aerosol alpha1-antitrypsin treatment for cystic fibrosis. Lancet1991; 337: 392–4.