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Karlovy40121890013359113148174234286341
a3402040013353
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Praha1860204001335893146206258
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Karlova,24832190013359113148174234286338361
Praha2867219001335993146207259
Genová3402700012165116172227274322
terapie691270001213382115164220244294
dle1013270001215780131
definice11722700012156107136163218241286335
Evropské1535270001214997130185242281328377
lékové1940270001212374121175222271
agentury22392700012149105155211243298330381
je2648270001212475
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genu11012840012156106162217
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genových33972980012156107162217265315359414
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a6433120012148
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poli4603260012157112136158
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svalovou2332326001213988136159214260314368
atrofii,2718326001214880112166195220243263
dále29993260012156103127176
genová31933260012155105160215261309
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primární500340001215689112194242273329351
poruchu8693400012156111144198243297351
syntézy1237340001213987143174223266315
biogenních1569340001215780134190240295349372416470
aminů20573400012147130152207261
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dekarboxylázy26373400012156106152201233289344389438462509552601
aromatických3256340001214880133216264295318362408458501555
L3403540012147
aminokyselin4043540012147129151206260306355394443467489543
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a13213540012148
genová13893540012155104159213259306
terapie1712354001213381113161216239288
pro2017354001215789142
metachromatickou21763540012183132165213256310342395478525556579623668721775
leukodystrofii.2968354001212373127172225281328366400431485513539561581
V35663540012155
USA36413540012164111170
je340368001212475
i4333680012124
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genová10183680012156106162217264313
terapie1349368001213383116164221244295
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Duchennovu18253680012167123168223273329384439486542
svalovou2386368001213988137160215263316372
dystrofii27763680012157104144178210265294320344
a31383680012148
pro3208368001215689144
X-vázanou3371368001215785133181225275330385440
adrenoleukodystrofii.3403820012148104136186241296319369424470524580628667700732787815841864885
V12423820012156
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již218338200121244789
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syndromu2927382001213987143199231285368423
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Leberovy340396001214796153203236290337387
hereditární7453960012155105137187244267299348380435458
optické12213960012154111144166211257305
neuropatie15443960012155105160192246303351383406456
a20173960012148
některých20873960012155105151187236268318362416
typů2521396001213382140194
mukopolysacharidóz,27333960012183137184237294349373420459508552607654686710766820862884
dále36343960012156104127177
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genová3844100012156106162217264312
terapie714410001213382115163220243293
u10254100012155
některých11014100012156106153189238270321364419
typů1538410001213383140195
pletencových1751410001215781131165214269314368415465509564
svalových2333410001213988136160215262312356411
dystrofií27624100012156103143176209263292318341
a31214100012148
u31904100012155
amyotrofické32674100012148131176230263295350379405449496544
laterální340424001212472105155189238261317341
sklerózy709424001214088113164197253295346
s10834240012140
vazbou1145424001214898141199256312
na14854240012156105
SOD116194240012147113179223
gen.18714240012156107164185
Efektivita2085424001214979130178214238286311344394
ale2507424001214973124
i26604240012123
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genové3404380012156107163218266315
terapie684438001213383116165222246296
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vždy1212438001214994150198
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k18824380012147
dané19504380012157105161212
diagnóze2190438001215780129185241296338388
i26074380012124
léčivému2652438001212474120143192241326381
přípravku.3061438001215790114171204253300348404425
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finanční340452001212954108155209253307329
nákladnost6864520012155102148172219274328382421454
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otázky1482452001215486134177222272
genové17714520012155104158212258306
terapie2094452001213381113161216239288
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na26484520012154101
celospolečenskou2766452001214492115169208263318341390435483537575621673727
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Klíčová340468001246190118163221273326
slova:686468001244472131183236261
genová9694690012156106162217264313
terapie,1303469001213382115164220244294314
virový1638469001214872105159206257
vektor,1916469001214897145180234267283
efektivita2221469001215079130177213236284308341390
léčby,2632469001212474120176222238
nežádoucí28924690012155106149199255310366411434
účinky.33474690012155100123178224275291
Gene3404960012468122182235
thepapy596496001243696150211264324376
of992496001245792
(selected)1104496001243273126155209255294347408438
neurological15634960012458112171210268296355414442488541569
diseases2153496001245987131185238282335380
According3405140012163108153209243301326383441
to811514001213389
the930514001213491143
definition11035140012158110141168225250284308365423
of1556514001215686
the1672514001213491143
European18455140012150106140196255308358415
Medicines22905140012182135194218265289346398439
Agency,27595140012163119172229276327344
gene31335140012157109167219
therapy3382514001213491143177228286334
is3746514001212465
a3405280012148
treatment409528001213366116164196280330386418
based8535280012157106145195253
on11325280012155110
the1268528001213389139
insertion1433528001212379118168201237260315371
of1830528001215584
a19405280012148
recombinant2009528001213383129182266323346401449505537
gene25725280012156106162212
into2810528001212479111165
a30015280012149
patient's30715280012157106138161211267299320358
body34515280012156112169217
for3694528001212984117
therapeutic,340542001213389140174223280332388422445490512
prophylactic,881542001215791146204261307331380425461485530551
or1461542001215689
diagnostic1579542001215780129185242297337372395440
purposes.20485420012158114147205261301352392414
In2491542001212480
recent2600542001213383130179236269
years,2897542001214898147179220242
there3168542001213389140173224
has34215420012156104144
been35945420012157109161217
a3405560012148
growing410556001215688143216240296352
number7905560012156111194251302335
of1154556001215584
clinical126655600121456992147170215264288
studies1582556001214074129186209260299
as1910556001214887
well20265560012173123147170
as2225556001214887
an23415560012148104
increasing2473556001212379124157207255294317373429
number29305560012156111194251302335
of3294556001215584
approved34065560012149106163196251298348405
gene3405700012156106162212
therapies,581570001213388138171220277300351390411
particularly10205700012157106139174198242297321369401425472
for1521570001212984117
rare1666570001213381114164
monogenic18585700012183139194249306357412435480
diseases.2366570001215780119170218257307347368
The27625700012150107157
first294757000121305688129162
examples3138570001215097145229285309360399
in3566570001212378
the3672570001213389139
field340584001212955105129185
of542584001215584
neurology6435840012155106160193247270325381429
include1089584001212478123146200256306
gene14135840012156105161211
therapy1641584001213388138171219275321
for1979584001212984117
spinal2113584001214096119174221244
muscular23755840012183137176221275299346378
atrophy,2771584001214880112166223279324339
gene31285840012156106161211
therapy3356584001213388138171219275321
for3694584001213084117
primary340598001215790113196244277327
disorders685598001215780119174207263313346387
of1090598001215483
biogenic1191598001215781136193243299322366
amine15755980012149132155210260
synthesis1853598001214088144177232282322345384
(aromatic2255598001212873105160244292324347392
L-amino2665598001214770118201224279334
acid3017598001214993116173
decarboxylase32085980012156106152201233290346391442466514553603
[AADC]340612001212886148214272302
deficiency),6606120012156106135161206228278333378427454472
and11506120012148102159
gene13266120012156106161211
therapy1555612001213387137170218274320
for1893612001212984116
metachromatic20276120012183133166215259314346400483531563586630
leukodystrophy.2675612001212373128175228285332371405437491548603649665
In3357612001212479
the3454612001213387138
United360961200121651191421753406260012150107
States,472626001215081130162212251272
gene7696260012156106162213
therapy1007626001213388138171220276323
has13556260012155103142
also1522626001214872111166
been17136260012157108159215
approved19536260012148104162195249296345403
for2381626001212984117
Duchenne25236260012166122167222273328384434
muscular29826260012183138177222277301349381
dystrophy33886260012157104144178210265322378423
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X-linked524640001215785110133188235284341
adrenoleukodystrophy.8896400012149105138188243298322373428475528586633673707739794851907952969
Clinical1882640001215881104159182227276299
studies2205640001214074129185209259298
are2527640001214981131
already2682640001214872104154202259306
underway30126400012156111168218251327375421
for3457640001213085117
gene35996400012156106162212
therapy340654001213388138171219275321
targeting679654001213382114169219253276330386
Dravet1083654001216699148194243276
syndrome,1377654001213987143199232286369420439
Leber's1834654001214797153204237256294
hereditary21496540012155105138188244267300349381431
optic25986540012155112145167212
neuropathy,28286540012155105160192247303352384439484500
and33466540012148103159
certain3523654001214593126162210233288
types340668001213383140191230
of588668001215584
mucopolysaccharidoses.68966800121831381832362933493734204595085525966516987317548108659049549941015
Additionally,17226680012161116173196228251306362409433456503519
gene22586680012156106162212
therapy2487668001213388139171219276322
is2827668001212362
being29076680012156107130185241
investigated3166668001212378124173212246269325372405454511
for3695668001212983116
some340682001214095178228
forms586682001212984117201239
of843682001215584
limb-girdle945682001212446129186219278301333389412462
muscular14256820012184138177222277300348380
dystrophies18236820012156104143177209264321377400450489
and23306820012148103160
for2507682001213084117
amyotrophic26426820012148131177230263296350407463486530
lateral3190682001212472104153186234258
sclerosis3465682001214085108158191245284307346
(ALS)340696001212889135186213
linked572696001212447102149198255
to846696001213387
the952696001213388139
SOD111106960012149118185232
gene.13616960012156106162212232
The16126960012150106157
effectiveness1788696001215079112162208243266315364419470509548
and23566960012148103160
side2535696001213962119169
effect2723696001215080112163208244
profile2986696001215790144173199223273
of3278696001215584
gene33816960012156106162212
therapy36126960012133891391723407100012148105151
are510710001214881131
always659710001214972145194239279
specific957710001213996147193216245271316
to1292710001213387
the1398710001213388138
particular15557100012157106138174197242297320368401
diagnosis1974710001215780128184239294333356395
and23887100012148104160
therapeutic2567710001213388139172220277328383416439484
product.3070710001215790144201256301336358
The34477100012149106156
high3622710001215578134190
financial340724001212955111159214259282330353
cost716724001214599138172
and9117240012148103160
ethical1094724001215084139162207255279
considerations1396724001214598154193216273323356404437460515571610
surrounding2029724001213994126159214269324381404459516
gene25687240012156106162212
therapy2803724001213388139172220277323
remain3149724001213383166214238293
subjects3465724001213994151174225270306346
for340738001212984117
broader478738001215790145194250301333
societal833738001213994140163213247295319
discussion.1173738001215780119164219258297320375431452
Key3407540012461113164
words:5257540012475133171232276302
gene8487550012156106162212
therapy,1082755001213388138171220276323339
viral1442755001214872104153176
vector,1639755001214898143179233265281
treatment1942755001213365115163196279329385418
effectiveness,2381755001215080112163208243267315364420470510549570
side2972755001214062119169
effects.3163755001215079112162208243284305
DECLARATIONS:4068273008648871301662142563043393604124625014570273008514
Declaration406828600874987120141179207245272292334376
of445828600874166
originality:45382860087406888131151193231252272299338358
The406829800853473109
manuscript418829800855791130168195227249266306329
is452829800851644
original45832980085386178117133172205222
and481629800853472112
has49392980085387299
not50502980085387699
been516129800853975110149
published532129800854078118135151178217252292
or406830900853862
submitted414530900852867108167184208234269310
elsewhere.44703090085355381117169209246269305320
Ethical406832200873764107127160198219
principles43013220087427090132165185229249288319
compliance:463532200873172134177198218256298331369388
The406833400853678116
authors420533400853475100142183208238
attest44643340085356087124154179
that466433400852465101126
their481133400852465103121146
study49783340085295596138173
was517333400855389118
approved531333400853477119144185220257300
by406834500854073
the41583450085236399
local42743450085165689124141
Ethical44323450085345898115147182199
Committee464834500854179139198215239265300337
and500134500853474114
is513234500851745
in519434500851656
compliance526634500853270130170188205239279311347
with40683560085537298140
human423635600854081143180221
studies44863560085285496139157195226
and474035600853577119
animal48883560085347695156193212
welfare512835600855391110133170196233
regulations53903560085236110414516420140683670085234080120148
of423636700853859
the43153670085236399
authors’44343670085347397136176200229244
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