Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M. Gene therapy comes of age. Science. 2018;359:eaan4672.

Article  PubMed  Google Scholar 

Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18:358–78.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Samulski RJ, Muzyczka N. AAV-mediated gene therapy for research and therapeutic purposes. Annu Rev Virol. 2014;1:427–51.

Article  PubMed  Google Scholar 

Yla-Herttuala S. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European Union. Mol Ther. 2012;20:1831–2.

Article  PubMed  PubMed Central  Google Scholar 

Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017;390:849–60.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl J Med. 2017;377:1713–22.

Article  CAS  PubMed  Google Scholar 

George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, et al. Hemophilia B gene therapy with a high-specific-activity Factor IX variant. N. Engl J Med. 2017;377:2215–27.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, et al. AAV5-Factor VIII gene transfer in severe Hemophilia A. N. Engl J Med. 2017;377:2519–30.

Article  CAS  PubMed  Google Scholar 

Keam SJ. Eladocagene Exuparvovec: First approval. Drugs. 2022;82:1427–32.

CAS  PubMed  Google Scholar 

Hoy SM. Delandistrogene Moxeparvovec: First approval. Drugs. 2023;83:1323–9.

Article  CAS  PubMed  Google Scholar 

Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol. 1996;70:8098–108.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Zhou S, Murphy JE, Escobedo JA, Dwarki VJ. Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates. Gene Ther. 1998;5:665–70.

Article  CAS  PubMed  Google Scholar 

Rivera VM, Ye X, Courage NL, Sachar J, Cerasoli F Jr., Wilson JM, et al. Long-term regulated expression of growth hormone in mice after intramuscular gene transfer. Proc Natl Acad Sci USA. 1999;96:8657–62.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Ye X, Rivera VM, Zoltick P, Cerasoli F Jr., Schnell MA, Gao G, et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science. 1999;283:88–91.

Article  CAS  PubMed  Google Scholar 

Rivera VM, Gao GP, Grant RL, Schnell MA, Zoltick PW, Rozamus LW, et al. Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood. 2005;105:1424–30.

Article  CAS  PubMed  Google Scholar 

Bohl D, Salvetti A, Moullier P, Heard JM. Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector. Blood. 1998;92:1512–7.

Article  CAS  PubMed  Google Scholar 

Chenuaud P, Larcher T, Rabinowitz JE, Provost N, Cherel Y, Casadevall N, et al. Autoimmune anemia in macaques following erythropoietin gene therapy. Blood. 2004;103:3303–4.

Article  CAS  PubMed  Google Scholar 

Zhong G, Wang H, Bailey CC, Gao G, Farzan M. Rational design of aptazyme riboswitches for efficient control of gene expression in mammalian cells. Elife. 2016;5:e18858.

Article  PubMed  PubMed Central  Google Scholar 

Luo L, Jea JD, Wang Y, Chao PW, Yen L. Control of mammalian gene expression by modulation of polyA signal cleavage at 5’ UTR. Nat Biotechnol. 2024;42:1454–66.

Monteys AM, Hundley AA, Ranum PT, Tecedor L, Muehlmatt A, Lim E, et al. Regulated control of gene therapies by drug-induced splicing. Nature. 2021;596:291–5.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Zhong G, Wang H, He W, Li Y, Mou H, Tickner ZJ, et al. A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo. Nat Biotechnol. 2020;38:169–75.

Article  CAS  PubMed  Google Scholar 

Fukunaga K, Dhamodharan V, Miyahira N, Nomura Y, Mustafina K, Oosumi Y, et al. Small-Molecule Aptamer for Regulating RNA Functions in Mammalian Cells and Animals. J Am Chem Soc. 2023;145:7820–8.

Theil D, Kuhle J, Brees D, Tritto E, Pognan F, Frieauff W, et al. Neurofilament light chain: a translational safety biomarker for drug-induced peripheral neurotoxicity. Toxicol Pathol. 2023;51:135–47.

Article  CAS  PubMed  Google Scholar 

Hastie E, Samulski RJ. Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success–a personal perspective. Hum Gene Ther. 2015;26:257–65.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Favre D, Blouin V, Provost N, Spisek R, Porrot F, Bohl D, et al. Lack of an immune response against the tetracycline-dependent transactivator correlates with long-term doxycycline-regulated transgene expression in nonhuman primates after intramuscular injection of recombinant adeno-associated virus. J Virol. 2002;76:11605–11.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Wang PR, Xu M, Toffanin S, Li Y, Llovet JM, Russell DW. Induction of hepatocellular carcinoma by in vivo gene targeting. Proc Natl Acad Sci USA. 2012;109:11264–9.

Article  CAS  PubMed  PubMed Central  Google Scholar 

David RM, Doherty AT. Viral vectors: the road to reducing genotoxicity. Toxicol Sci. 2017;155:315–25.

Article  CAS  PubMed  Google Scholar 

Wilton-Clark H, Yokota T. Recent trends in Antisense therapies for Duchenne Muscular Dystrophy. Pharmaceutics. 2023;15:778.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Woo S, Jusko WJ. Interspecies comparisons of pharmacokinetics and pharmacodynamics of recombinant human erythropoietin. Drug Metab Dispos. 2007;35:1672–8.

Article  CAS  PubMed  Google Scholar 

Blum S, Shapir N, Miari R, Lerner B, Koren B, Doenyas-Barak K, et al. TARGT gene therapy platform for correction of anemia in end-stage renal disease. N. Engl J Med. 2017;376:189–91.

Article  PubMed  Google Scholar 

Bunn HF. Erythropoietin. Cold Spring Harb Perspect Med. 2013;3:a011619.

Article  PubMed  PubMed Central  Google Scholar 

Fang J, Wang J, Wang Y, Liu X, Chen B, Zou W. Ribo-On and Ribo-Off tools using a self-cleaving ribozyme allow manipulation of endogenous gene expression in C. elegans. Commun Biol. 2023;6:816.

Article  CAS  PubMed  PubMed Central