ShawWL. Selective service litigation and the 1967 statute. Mil L Rev, 1970; 48:33–89.
2.
CrystalRG, ShafritzDA, PrichardPM, et al.Initial dipeptide formation in hemoglobin biosynthesis. Proc Natl Acad Sci U S A, 1971; 68:1810–1814.
3.
FinchRH. Statement of organization, functions, and delegations of authority. Federal Register, 1969; 34:18395.
4.
HurdSS, LenfantC. NHLBI: fifty years of achievement in pulmonary biology and medicine. Am J Respir Crit Care Med, 1998; 157:S168–171.
5.
BrantlyM, NukiwaT, CrystalRG. Molecular basis of alpha-1-antitrypsin deficiency. Am J Med, 1988; 84:13–31.
6.
HubbardRC, CrystalRG. Alpha-1-antitrypsin augmentation therapy for alpha-1-antitrypsin deficiency. Am J Med, 1988; 84:52–62.
7.
GadekJE, FellsGA, ZimmermanRL, et al.Antielastases of the human alveolar structures. Implications for the protease–antiprotease theory of emphysema. J Clin Invest, 1981; 68:889–898.
8.
GadekJE, KleinHG, HollandPV, et al.Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease–antiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Invest, 1981; 68:1158–1165.
9.
WewersMD, CasolaroMA, SellersSE, et al.Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med, 1987; 316:1055–1062.
10.
StrangeC. Anti-proteases and alpha-1 antitrypsin augmentation therapy. Respir Care, 2018; 63:690–698.
11.
ChapmanKR, BurdonJG, PiitulainenE, et al.Intravenous augmentation treatment and lung density in severe alpha1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial. Lancet, 2015; 386:360–368.
12.
McElvaneyNG, BurdonJ, HolmesM, et al.Long-term efficacy and safety of alpha1 proteinase inhibitor treatment for emphysema caused by severe alpha1 antitrypsin deficiency: an open-label extension trial (RAPID-OLE). Lancet Respir Med, 2017; 5:51–60.
13.
HubbardRC, CrystalRG. Strategies for aerosol therapy of alpha 1-antitrypsin deficiency by the aerosol route. Lung, 1990; 168Suppl:565–578.
14.
YuSF, von RudenT, KantoffPW, et al.Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc Natl Acad Sci U S A, 1986; 83:3194–3198.
15.
GarverRI Jr, ChytilA, KarlssonS, et al.Production of glycosylated physiologically “normal” human alpha 1-antitrypsin by mouse fibroblasts modified by insertion of a human alpha 1-antitrypsin cDNA using a retroviral vector. Proc Natl Acad Sci U S A, 1987; 84:1050–1054.
16.
GarverRI Jr, ChytilA, CourtneyM, et al.Clonal gene therapy: transplanted mouse fibroblast clones express human alpha 1-antitrypsin gene in vivo. Science, 1987; 237:762–764.
17.
WivelNA. Historical perspectives pertaining to the NIH Recombinant DNA Advisory Committee. Hum Gene Ther, 2014; 25:19–24.
18.
GilardiP, CourtneyM, PaviraniA, et al.Expression of human alpha 1-antitrypsin using a recombinant adenovirus vector. FEBS Lett, 1990; 267:60–62.
19.
RosenfeldMA, SiegfriedW, YoshimuraK, et al.Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science, 1991; 252:431–434.
20.
KeremB, RommensJM, BuchananJA, et al.Identification of the cystic fibrosis gene: genetic analysis. Science, 1989; 245:1073–1080.
21.
RiordanJR, RommensJM, KeremB, et al.Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science, 1989; 245:1066–1073.
22.
RommensJM, IannuzziMC, KeremB, et al.Identification of the cystic fibrosis gene: chromosome walking and jumping. Science, 1989; 245:1059–1065.
23.
RosenfeldMA, YoshimuraK, TrapnellBC, et al.In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell, 1992; 68:143–155.
24.
MurraryBE. Minutes of the RecombinantDNA advisory committee, December 3–4, 1992. https://osp.od.nih.gov/wp-content/uploads/2014/03/12-34-92.pdf (last accessed March9, 2020).
25.
AngierN.Gene therapy begins for fatal lung disease. The New York Times, April 20, 1993, p. 5.
26.
ZabnerJ, CoutureLA, GregoryRJ, et al.Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell, 1993; 75:207–216.
27.
CrystalRG, McElvaneyNG, RosenfeldMA, et al.Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet, 1994; 8:42–51.
28.
GoldmanMJ, YangY, WilsonJM. Gene therapy in a xenograft model of cystic fibrosis lung corrects chloride transport more effectively than the sodium defect. Nat Genet, 1995; 9:126–131.
29.
GrubbBR, PicklesRJ, YeH, et al.Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans. Nature, 1994; 371:802–806.
30.
HarveyBG, LeopoldPL, HackettNR, et al.Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus. J Clin Invest, 1999; 104:1245–1255.
31.
ZabnerJ, RamseyBW, MeekerDP, et al.Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis. J Clin Invest, 1996; 97:1504–1511.
32.
YangY, NunesFA, BerencsiK, et al.Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nat Genet, 1994; 7:362–369.
33.
ArmentanoD, ZabnerJ, SacksC, et al.Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J Virol, 1997; 71:2408–2416.
34.
BruderJT, JieT, McVeyDL, et al.Expression of gp19K increases the persistence of transgene expression from an adenovirus vector in the mouse lung and liver. J Virol, 1997; 71:7623–7628.
35.
KaplanJM, ArmentanoD, SparerTE, et al.Characterization of factors involved in modulating persistence of transgene expression from recombinant adenovirus in the mouse lung. Hum Gene Ther, 1997; 8:45–56.
36.
ChirmuleN, HughesJV, GaoGP, et al.Role of E4 in eliciting CD4 T-cell and B-cell responses to adenovirus vectors delivered to murine and nonhuman primate lungs. J Virol, 1998; 72:6138–6145.
37.
HubbardRC, McElvaneyNG, BirrerP, et al.A preliminary study of aerosolized recombinant human deoxyribonuclease I in the treatment of cystic fibrosis. N Engl J Med, 1992; 326:812–815.
38.
LeeLY, PatelSR, HackettNR, et al.Focal angiogen therapy using intramyocardial delivery of an adenovirus vector coding for vascular endothelial growth factor 121. Ann Thorac Surg, 2000; 69:14–23; discussion 23–24.
39.
LeottaE, PatejunasG, MurphyG, et al.Gene therapy with adenovirus-mediated myocardial transfer of vascular endothelial growth factor 121 improves cardiac performance in a pacing model of congestive heart failure. J Thorac Cardiovasc Surg, 2002; 123:1101–1113.
40.
MackCA, MagovernCJ, BudenbenderKT, et al.Salvage angiogenesis induced by adenovirus-mediated gene transfer of vascular endothelial growth factor protects against ischemic vascular occlusion. J Vasc Surg, 1998; 27:699–709.
41.
MackCA, PatelSR, SchwarzEA, et al.Biologic bypass with the use of adenovirus-mediated gene transfer of the complementary deoxyribonucleic acid for vascular endothelial growth factor 121 improves myocardial perfusion and function in the ischemic porcine heart. J Thorac Cardiovasc Surg, 1998; 115:168–176; discussion 176–167.
42.
MagovernCJ, MackCA, ZhangJ, et al.Direct in vivo gene transfer to canine myocardium using a replication-deficient adenovirus vector. Ann Thorac Surg, 1996; 62:425–433; discussion 433–424.
43.
MagovernCJ, MackCA, ZhangJ, et al.Regional angiogenesis induced in nonischemic tissue by an adenoviral vector expressing vascular endothelial growth factor. Hum Gene Ther, 1997; 8:215–227.
44.
PatelSR, LeeLY, MackCA, et al.Safety of direct myocardial administration of an adenovirus vector encoding vascular endothelial growth factor 121. Hum Gene Ther, 1999; 10:1331–1348.
45.
RosengartTK, LeeLY, PatelSR, et al.Six-month assessment of a Phase I trial of angiogenic gene therapy for the treatment of coronary artery disease using direct intramyocardial administration of an adenovirus vector expressing the VEGF121 cDNA. Ann Surg, 1999; 230:466–470; discussion 470–462.
46.
RosengartTK, LeeLY, PatelSR, et al.Angiogenesis gene therapy: Phase I assessment of direct intramyocardial administration of an adenovirus vector expressing VEGF121 cDNA to individuals with clinically significant severe coronary artery disease. Circulation, 1999; 100:468–474.
47.
SchalchP, RahmanGF, PatejunasG, et al.Adenoviral-mediated transfer of vascular endothelial growth factor 121 cDNA enhances myocardial perfusion and exercise performance in the nonischemic state. J Thorac Cardiovasc Surg, 2004; 127:535–540.
48.
StewartKJ, BadenhopD, BrubakerPH, et al.Cardiac rehabilitation following percutaneous revascularization, heart transplant, heart valve surgery, and for chronic heart failure. Chest, 2003; 123:2104–2111.
49.
StewartDJ, HiltonJD, ArnoldJM, et al.Angiogenic gene therapy in patients with nonrevascularizable ischemic heart disease: a phase 2 randomized, controlled trial of AdVEGF(121) (AdVEGF121) versus maximum medical treatment. Gene Ther, 2006; 13:1503–1511.
50.
AmanoH, HackettNR, KanerRJ, et al.Alteration of splicing signals in a genomic/cDNA hybrid VEGF gene to modify the ratio of expressed VEGF isoforms enhances safety of angiogenic gene therapy. Mol Ther, 2005; 12:716–724.
51.
WhitlockPR, HackettNR, LeopoldPL, et al.Adenovirus-mediated transfer of a minigene expressing multiple isoforms of VEGF is more effective at inducing angiogenesis than comparable vectors expressing individual VEGF cDNAs. Mol Ther, 2004; 9:67–75.
52.
SondhiD, HackettNR, ApblettRL, et al.Feasibility of gene therapy for late neuronal ceroid lipofuscinosis. Arch Neurol, 2001; 58:1793–1798.
53.
WorgallS, SondhiD, HackettNR, et al.Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther, 2008; 19:463–474.
54.
ZhaoL, GottesdienerAJ, ParmarM, et al.Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models. Neurobiol Aging, 2016; 44:159–172.
55.
RosenbergJB, KaplittMG, DeBP, et al.AAVrh.10-mediated APOE2 central nervous system gene therapy for APOE4-associated Alzheimer's disease. Hum Gene Ther Clin Dev, 2018; 29:24–47.
56.
SacramentoCB, SondhiD, RosenbergJB, et al.Anti-phospho-tau gene therapy for chronic traumatic encephalopathy. Hum Gene Ther, 2020; 31:57–69.
57.
DeB, HeguyA, LeopoldPL, et al.Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin. Mol Ther, 2004; 10:1003–1010.
58.
MatsumuraY, LiN, AlwaseemH, et al.Systemic adeno-associated virus-mediated gene therapy prevents the multi-organ disorders associated with aldehyde dehydrogenase 2 deficiency and chronic ethanol ingestion. Hum Gene Ther, 2020; 31:163–182.
59.
MatsumuraY, StilesKM, ReidJ, et al.Gene therapy correction of aldehyde dehydrogenase 2 deficiency. Mol Ther Methods Clin Dev, 2019; 15:72–82.
60.
SalamiCO, RosenbergJB, JacksonKL, et al.Stress-induced cardiac mouse model of Friedreich's ataxia corrected by AAV-mediated gene therapy. Mol Ther, 2018; 26:348.
61.
SondhiD, RosenbergJB, DeBP, et al.Long-term toxicology evaluation of AAVrh.10hARSA administration to the CNS of nonhuman primates to treat metachromatic leukodystrophy. Mol Ther, 2016; 24:S146.
62.
SondhiD, ScottEC, ChenA, et al.Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene. Hum Gene Ther, 2014; 25:223–239.
63.
MarkmannS, J Christie-ReidJ, RosenbergJB, et al.Attenuation of the Niemann–Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2. Exp Neurol, 2018; 306:22–33.
64.
PagovichOE, NagS, StilesKM, et al.Gene therapy for chronic eosinophilic leukemia. Mol Ther, 2019; 27:166.
65.
QiuT, ChiuchioloMJ, WhaleyAS, et al.Gene therapy for C1 esterase inhibitor deficiency in a murine model of hereditary angioedema. Allergy, 2019; 74:1081–1089.
66.
SosulskiML, StilesKM, DeBP, et al.Assessment of a second generation, oxidation resistant gene therapy for alpha 1-antitrypsin deficiency. Mol Ther, 2019; 27:43.
67.
HicksMJ, ChiuchioloMJ, BallonD, et al.Anti-epidermal growth factor receptor gene therapy for glioblastoma. PLoS One, 2016; 11:e0162978.
68.
HicksMJ, FunatoK, WangL, et al.Genetic modification of neurons to express bevacizumab for local anti-angiogenesis treatment of glioblastoma. Cancer Gene Ther, 2015; 22:1–8.
69.
XieY, HicksMJ, KaminskySM, et al.AAV-mediated persistent bevacizumab therapy suppresses tumor growth of ovarian cancer. Gynecol Oncol, 2014; 135:325–332.
70.
WatanabeM, BoyerJL, CrystalRG. AAVrh.10-mediated genetic delivery of bevacizumab to the pleura to provide local anti-VEGF to suppress growth of metastatic lung tumors. Gene Ther, 2010; 17:1042–1051.
71.
WangG, QiuJ, WangR, et al.Persistent expression of biologically active anti-HER2 antibody by AAVrh.10-mediated gene transfer. Cancer Gene Ther, 2010; 17:559–570.
72.
HicksMJ, RosenbergJB, DeBP, et al.AAV-directed persistent expression of a gene encoding anti-nicotine antibody for smoking cessation. Sci Transl Med, 2012; 4:140ra187.
73.
WeeS, HicksMJ, DeBP, et al.Novel cocaine vaccine linked to a disrupted adenovirus gene transfer vector blocks cocaine psychostimulant and reinforcing effects. Neuropsychopharmacology, 2012; 37:1083–1091.
74.
RosenbergJB, HicksMJ, DeBP, et al.AAVrh.10-mediated expression of an anti-cocaine antibody mediates persistent passive immunization that suppresses cocaine-induced behavior. Hum Gene Ther, 2012; 23:451–459.
75.
DeBP, PagovichOE, HicksMJ, et al.Disrupted adenovirus-based vaccines against small addictive molecules circumvent anti-adenovirus immunity. Hum Gene Ther, 2013; 24:58–66.
76.
RosenbergJB, DeBP, HicksMJ, et al.Suppression of nicotine-induced pathophysiology by an adenovirus hexon-based antinicotine vaccine. Hum Gene Ther, 2013; 24:595–603.
77.
HicksMJ, KaminskySM, DeBP, et al.Fate of systemically administered cocaine in nonhuman primates treated with the dAd5GNE anti-cocaine vaccine. Hum Gene Ther Clin Dev, 2014; 25:40–49.
78.
TanY, HackettNR, BoyerJL, et al.Protective immunity evoked against anthrax lethal toxin after a single intramuscular administration of an adenovirus-based vaccine encoding humanized protective antigen. Hum Gene Ther, 2003; 14:1673–1682.
79.
KasuyaK, BoyerJL, TanY, et al.Passive immunotherapy for anthrax toxin mediated by an adenovirus expressing an anti-protective antigen single-chain antibody. Mol Ther, 2005; 11:237–244.
80.
HashimotoM, BoyerJL, HackettNR, et al.Induction of protective immunity to anthrax lethal toxin with a nonhuman primate adenovirus-based vaccine in the presence of preexisting anti-human adenovirus immunity. Infect Immun, 2005; 73:6885–6891.
81.
ChiuchioloMJ, BoyerJL, KrauseA, et al.Protective immunity against respiratory tract challenge with Yersinia pestis in mice immunized with an adenovirus-based vaccine vector expressing V antigen. J Infect Dis, 2006; 194:1249–1257.
82.
DeBP, HackettNR, CrystalRG, et al.Rapid/sustained anti-anthrax passive immunity mediated by co-administration of Ad/AAV. Mol Ther, 2008; 16:203–209.
83.
Sofer-PodestaC, AngJ, HackettNR, et al.Adenovirus-mediated delivery of an anti-V antigen monoclonal antibody protects mice against a lethal Yersinia pestis challenge. Infect Immun, 2009; 77:1561–1568.
84.
BoyerJL, Sofer-PodestaC, AngJ, et al.Protective immunity against a lethal respiratory Yersinia pestis challenge induced by V antigen or the F1 capsular antigen incorporated into adenovirus capsid. Hum Gene Ther, 2010; 21:891–901.
85.
KoobG, HicksMJ, WeeS, et al.Anti-cocaine vaccine based on coupling a cocaine analog to a disrupted adenovirus. CNS Neurol Disord Drug Targets, 2011; 10:899–904.
86.
HicksMJ, DeBP, RosenbergJB, et al.Cocaine analog coupled to disrupted adenovirus: a vaccine strategy to evoke high-titer immunity against addictive drugs. Mol Ther, 2011; 19:612–619.
87.
MaozA, HicksMJ, VallabhjosulaS, et al.Adenovirus capsid-based anti-cocaine vaccine prevents cocaine from binding to the nonhuman primate CNS dopamine transporter. Neuropsychopharmacology, 2013; 38:2170–2178.