Human Gene Therapy - April 2023 - 302

302
RANA ET AL.
61. Jungmann A, Muller O, Rapti K. Cell-based
measurement of neutralizing antibodies against
adeno-associated virus (AAV). Methods Mol Biol
2017;1521:109-126; doi: 10.1007/978-1-49396588-5_7
62.
Steven W, Pipe MR, Key NS, et al. First data from
the phase 3 HOPE-B gene therapy trial: Efficacy
and safety of etranacogene dezaparvovec (AAV5Padua
hFIX variant; AMT-061) in adults with severe
or moderate-severe hemophilia B treated irrespective
of pre-existing anti-capsid neutralizing
antibodies. Blood 2020;136:LBA-6; doi: 10.1182/
blood-2020-143560
63. Sen D, Balakrishnan B, Gabriel N, et al. Improved
adeno-associated virus (AAV) serotype 1 and 5
vectors for gene therapy. Sci Rep 2013;3:1832;
doi: 10.1038/srep01832
64. Agbandje-McKenna M, Kleinschmidt J. AAV capsid
structure and cell interactions. Methods Mol Biol
2011;807:47-92; doi: 10.1007/978-1-61779-370-7_3
65. Mietzsch M, Jose A, Chipman P, et al. Completion
of the AAV structural atlas: Serotype capsid
structures reveals clade-specific features. Viruses
2021;13(1):101; doi: 10.3390/v13010101
66. Govindasamy L, DiMattia MA, Gurda BL, et al.
Structural insights into adeno-associated virus
serotype 5. J Virol 2013;87(20):11187-11199; doi:
10.1128/JVI.00867-13
67. Lerch TF, Xie Q, Chapman MS. The structure of
adeno-associated virus serotype 3B (AAV-3B): Insights
into receptor binding and immune evasion.
Virology 2010;403(1):26-36; doi: 10.1016/
j.virol.2010.03.027
68. FDA. Toxicity Risks of Adeno-associated Virus
(AAV) Vectors for Gene Therapy (GT). Cellular,
Tissue, and Gene Therapies Advisory Committee
(CTGTAC) Meeting #70, Food and Drug Administration,
United States, 2021.
69. Chand DH, Zaidman C, Arya K, et al. Thrombotic
microangiopathy following onasemnogene abeparvovec
for spinal muscular atrophy: A case series.
J Pediatr 2021;231:265-268; doi: 10.1016/
j.jpeds.2020.11.054
70. Bonnemann CB, Braun S, Morris C, et al. A Collaborative
Analysis by Clinical Trial Sponsors and
Academic Experts of Anti-Transgene SAES in
Studies of Gene Therapy for DMD. In: 2022
Muscular Dystrophy Association conference 2022.
Conference abstract.
71. Konkle BA, Walsh CE, Escobar MA, et al. BAX 335
hemophilia B gene therapy clinical trial results:
Potential impact of CpG sequences on gene expression.
Blood 2021;137(6):763-774; doi:
10.1182/blood.2019004625
72. Hamilton BA, Wright JF. Challenges posed by
immune responses to AAV vectors: Addressing
root causes. Front Immunol 2021;12:675897; doi:
10.3389/fimmu.2021.675897
73. Paulk NK, Pekrun K, Zhu E, et al. Bioengineered
AAV capsids with combined high human liver
transduction in vivo and unique humoral seroreactivity.
Mol Ther 2018;26(1):289-303; doi:
10.1016/j.ymthe.2017.09.021
74. Grimm D, Lee JS, Wang L, et al. In vitro and
in vivo gene therapy vector evolution via multispecies
interbreeding and retargeting of adenoassociated
viruses. J Virol 2008;82(12):5887-
5911; doi: 10.1128/JVI.00254-08
75. Ling C, Lu Y, Kalsi JK, et al. Human hepatocyte
growth factor receptor is a cellular coreceptor for
adeno-associated virus serotype 3. Hum Gene
Ther 2010;21(12):1741-1747; doi: 10.1089/
hum.2010.075
76. Govindasamy L, Padron E, McKenna R, et al.
Structurally mapping the diverse phenotype of
adeno-associated virus serotype 4. J Virol 2006;
80(23):11556-11570; doi: 10.1128/JVI.01536-06
77. Tseng YS, Agbandje-McKenna M. Mapping the
AAV capsid host antibody response toward the
development of second generation gene delivery
vectors. Front Immunol 2014;5:9; doi: 10.3389/
fimmu.2014.00009
78. Havlik LP, Simon KE, Smith JK, et al. Coevolution of
adeno-associated virus capsid antigenicity and tropism
through a structure-guided approach. J Virol
2020;94(19):e00976-20; doi: 10.1128/JVI.00976-20
79. Perocheau DP, Cunningham S, Lee J, et al. Agerelated
seroprevalence of antibodies against AAVLK03
in a UK population cohort. Hum Gene Ther
2019;30(1):79-87; doi: 10.1089/hum.2018.098
80. van der Marel S, Comijn EM, Verspaget HW, et al.
Neutralizing antibodies against adeno-associated
viruses in inflammatory bowel disease patients:
implications for gene therapy. Inflamm Bowel Dis
2011;17(12):2436-2442; doi: 10.1002/ibd.21673
81. Pipe SW, Recht M, Key NS, et al. Durability of
bleeding protection and factor IX activity levels
are demonstrated in individuals with and without
adeno-associated virus serotype 5 neutralizing
antibodies (titers <1:700) with comparable safety
in the phase 3 HOPE-B clinical trial of etranacogene
dezaparvovec gene therapy for hemophilia B.
Blood 2022;140:4904-4906; doi: 10.1182/blood2022-166745
82.
Majowicz A, Nijmeijer B, Lampen MH, et al.
Therapeutic hFIX activity achieved after single
AAV5-hFIX treatment in hemophilia B patients and
NHPs with pre-existing anti-AAV5 NABs. Mol
Ther Methods Clin Dev 2019;14:27-36; doi:
10.1016/j.omtm.2019.05.009
83. Nonnenmacher M, Wang W, Child MA, et al.
Rapid evolution of blood-brain-barrier-penetrating
AAV capsids by RNA-driven biopanning. Mol Ther
Methods Clin Dev 2021;20:366-378; doi: 10.1016/
j.omtm.2020.12.006
84. Westhaus A, Cabanes-Creus M, Jonker T, et al. AAVp40
bioengineering platform for variant selection
basedon transgeneexpression. HumGene Ther 2022;
33(11-12):664-682; doi: 10.1089/hum.2021.278
Received for publication August 28, 2022;
accepted after revision February 25, 2023.
Published online: March 23, 2023.
http://dx.doi.org/10.1016/j.jpeds.2020.11.054 http://dx.doi.org/10.1016/j.jpeds.2020.11.054 http://dx.doi.org/10.1007/978-1-4939-6588-5_7 http://dx.doi.org/10.1128/JVI.00976-20 http://dx.doi.org/10.1007/978-1-4939-6588-5_7 http://dx.doi.org/10.1089/hum.2018.098 http://dx.doi.org/10.1182/blood-2020-143560 http://dx.doi.org/10.1182/blood-2020-143560 http://dx.doi.org/10.1002/ibd.21673 http://dx.doi.org/10.1182/blood.2019004625 http://dx.doi.org/10.1038/srep01832 http://dx.doi.org/10.3389/fimmu.2021.675897 http://dx.doi.org/10.1007/978-1-61779-370-7_3 http://dx.doi.org/10.1182/blood-2022-166745 http://dx.doi.org/10.1182/blood-2022-166745 http://dx.doi.org/10.1016/j.ymthe.2017.09.021 http://dx.doi.org/10.3390/v13010101 http://dx.doi.org/10.1128/JVI.00254-08 http://dx.doi.org/10.1016/j.omtm.2019.05.009 http://dx.doi.org/10.1128/JVI.00867-13 http://dx.doi.org/10.1016/j.omtm.2020.12.006 http://dx.doi.org/10.1089/hum.2010.075 http://dx.doi.org/10.1016/j.omtm.2020.12.006 http://dx.doi.org/10.1016/j.virol.2010.03.027 http://dx.doi.org/10.1089/hum.2010.075 http://dx.doi.org/10.1016/j.virol.2010.03.027 http://dx.doi.org/10.1089/hum.2021.278 http://dx.doi.org/10.1128/JVI.01536-06 http://dx.doi.org/10.3389/fimmu.2014.00009 http://dx.doi.org/10.3389/fimmu.2014.00009

Human Gene Therapy - April 2023

Table of Contents for the Digital Edition of Human Gene Therapy - April 2023

Contents
Human Gene Therapy - April 2023 - CT1
Human Gene Therapy - April 2023 - CT2
Human Gene Therapy - April 2023 - Cover1
Human Gene Therapy - April 2023 - Cover2
Human Gene Therapy - April 2023 - 239
Human Gene Therapy - April 2023 - 240
Human Gene Therapy - April 2023 - 241
Human Gene Therapy - April 2023 - 242
Human Gene Therapy - April 2023 - 243
Human Gene Therapy - April 2023 - 244
Human Gene Therapy - April 2023 - Contents
Human Gene Therapy - April 2023 - 246
Human Gene Therapy - April 2023 - 247
Human Gene Therapy - April 2023 - 248
Human Gene Therapy - April 2023 - 249
Human Gene Therapy - April 2023 - 250
Human Gene Therapy - April 2023 - 251
Human Gene Therapy - April 2023 - 252
Human Gene Therapy - April 2023 - 253
Human Gene Therapy - April 2023 - 254
Human Gene Therapy - April 2023 - 255
Human Gene Therapy - April 2023 - 256
Human Gene Therapy - April 2023 - 257
Human Gene Therapy - April 2023 - 258
Human Gene Therapy - April 2023 - 259
Human Gene Therapy - April 2023 - 260
Human Gene Therapy - April 2023 - 261
Human Gene Therapy - April 2023 - 262
Human Gene Therapy - April 2023 - 263
Human Gene Therapy - April 2023 - 264
Human Gene Therapy - April 2023 - 265
Human Gene Therapy - April 2023 - 266
Human Gene Therapy - April 2023 - 267
Human Gene Therapy - April 2023 - 268
Human Gene Therapy - April 2023 - 269
Human Gene Therapy - April 2023 - 270
Human Gene Therapy - April 2023 - 271
Human Gene Therapy - April 2023 - 272
Human Gene Therapy - April 2023 - 273
Human Gene Therapy - April 2023 - 274
Human Gene Therapy - April 2023 - 275
Human Gene Therapy - April 2023 - 276
Human Gene Therapy - April 2023 - 277
Human Gene Therapy - April 2023 - 278
Human Gene Therapy - April 2023 - 279
Human Gene Therapy - April 2023 - 280
Human Gene Therapy - April 2023 - 281
Human Gene Therapy - April 2023 - 282
Human Gene Therapy - April 2023 - 283
Human Gene Therapy - April 2023 - 284
Human Gene Therapy - April 2023 - 285
Human Gene Therapy - April 2023 - 286
Human Gene Therapy - April 2023 - 287
Human Gene Therapy - April 2023 - 288
Human Gene Therapy - April 2023 - 289
Human Gene Therapy - April 2023 - 290
Human Gene Therapy - April 2023 - 291
Human Gene Therapy - April 2023 - 292
Human Gene Therapy - April 2023 - 293
Human Gene Therapy - April 2023 - 294
Human Gene Therapy - April 2023 - 295
Human Gene Therapy - April 2023 - 296
Human Gene Therapy - April 2023 - 297
Human Gene Therapy - April 2023 - 298
Human Gene Therapy - April 2023 - 299
Human Gene Therapy - April 2023 - 300
Human Gene Therapy - April 2023 - 301
Human Gene Therapy - April 2023 - 302
Human Gene Therapy - April 2023 - 303
Human Gene Therapy - April 2023 - 304
Human Gene Therapy - April 2023 - 305
Human Gene Therapy - April 2023 - 306
Human Gene Therapy - April 2023 - 307
Human Gene Therapy - April 2023 - 308
Human Gene Therapy - April 2023 - 309
Human Gene Therapy - April 2023 - 310
Human Gene Therapy - April 2023 - 311
Human Gene Therapy - April 2023 - 312
Human Gene Therapy - April 2023 - 313
Human Gene Therapy - April 2023 - 314
Human Gene Therapy - April 2023 - 315
Human Gene Therapy - April 2023 - 316
Human Gene Therapy - April 2023 - 317
Human Gene Therapy - April 2023 - 318
Human Gene Therapy - April 2023 - 319
Human Gene Therapy - April 2023 - 320
Human Gene Therapy - April 2023 - 321
Human Gene Therapy - April 2023 - 322
Human Gene Therapy - April 2023 - 323
Human Gene Therapy - April 2023 - 324
Human Gene Therapy - April 2023 - 325
Human Gene Therapy - April 2023 - 326
Human Gene Therapy - April 2023 - 327
Human Gene Therapy - April 2023 - 328
Human Gene Therapy - April 2023 - 329
Human Gene Therapy - April 2023 - 330
Human Gene Therapy - April 2023 - 331
Human Gene Therapy - April 2023 - 332
Human Gene Therapy - April 2023 - 333
Human Gene Therapy - April 2023 - 334
Human Gene Therapy - April 2023 - 335
Human Gene Therapy - April 2023 - 336
Human Gene Therapy - April 2023 - 337
Human Gene Therapy - April 2023 - 338
Human Gene Therapy - April 2023 - 339
Human Gene Therapy - April 2023 - 340
Human Gene Therapy - April 2023 - 341
Human Gene Therapy - April 2023 - 342
Human Gene Therapy - April 2023 - Cover3
Human Gene Therapy - April 2023 - Cover4
https://www.nxtbookmedia.com