HUE031156T2 - Génterápia amiotróf laterális szklerózis és egyéb gerincvelõ-betegségek ellen - Google Patents

Génterápia amiotróf laterális szklerózis és egyéb gerincvelõ-betegségek ellen Download PDF

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HUE031156T2
HUE031156T2 HUE07795835A HUE07795835A HUE031156T2 HU E031156 T2 HUE031156 T2 HU E031156T2 HU E07795835 A HUE07795835 A HU E07795835A HU E07795835 A HUE07795835 A HU E07795835A HU E031156 T2 HUE031156 T2 HU E031156T2
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recombinant
viral vector
brain
vector
transgene
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HUE07795835A
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James Dodge
Lamya Shihabuddin
Catherine R O'riordan
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Genzyme Corp
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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Claims (7)

  1. GÉNTERÁPIA AMIOTRÓF LATERÁLIS SZKLERÓZ1S ÉS EGYÉB GERÍNVELÖ-BETEGSÉGEK ELLEN S*al>ad&!sii tgéövpenRík
    1, Transzgént .tartalmazó' rekombináns «etiroJrói: virnsvektor -a transzgén termőkét egy Személy gerincvelőjébe eélbajnti&tő eljárásban történő alkalmazásra, ahol az eljárás tartalmazza a virusvektor beadását legalább egy agykamráiba, miáltal a tramzgén expresszálodik és az expresszi:!!ttanszgémermék a gerincvelőbe jut, ahol: a virusvektor adeno-asszoeíáit virnsvektor (AA V) és a transzáén SMN-1.
  2. 2. Az 1. igénypont szerinti rekombináns neurotrőf virnsvektor az ott eljárás tartalmazza a virnsvektor beadását közvetlenül agykamníba történő mjektälSsÄ 5. A 2. igénypont szerinti rekombináns nemtixMvírasvektor az ott megbatározott alkalmazásra, ahol az eijítras tartalmazza a virnsvektor beadását közvetlenül laterális agykamrába történő injekiálással 4. A 2. igénypont szerinti rekombináns neurotrói' virusvektor az ott meghatározott alkalmazásra, aboi az eljárás tartalmazza a virnsvektor beadásál közvetlenül a negyedik agykamrába történő injektálással.
  3. 5. Az, i. igénypont szerinti rekombináns nenrotröf vlmsvektor az ott meghatározott alkaímazásrái bböl a transzgén további, a következők alkotta csoportból választott proteint is exprssszál: inzulin növekedés íáktor 1 (IGF-1), kalbindin- D28. parvalbutnin, HIFl-alfe, SÏRT-2, VEGE, SMN-2, CNTF {cilutris neurotrói' faktor), Sonic Hedgehog (SHH>. entropoietin (EFO), lizil-oxidáz (LOX), progranulím prolaktin, ghrelin, neuroserpin, angio-genin és placentakiktogén, Ő. Az 1. igénypont szerinti rekombináns alkalmazásra, öböl a szentélynek spinális izomattóflája vart,
  4. 7. Az Is igénypont szerinti rekombináns neurotrőf' virmsvektör az ott meghatározott alkalmazásra, ahol a személy emlős. 8. A 7, igénypont szerinti rekombináns neuron of virusvektor ázott meghatározott alkalmazásra, ahol az emlős a következők alkotta csoportból választott: rágcsáló, egerl'orata, majom, emberi 9. A» 1, igénypont szerimi rekombináns neurotrőf vírusvektor az otf meghatározott alkalmazásra, aböl a személy bntbáöipáelens,
  5. 10. SMN'Let kódoló iranszgéitl tartalmazó, rekombináns A AV-virus vektor SkTM-l-et spináits jzomat-róftában: szén védő személy gerincvelőjébe eélbajutiaíó eljárásban történő alkalmazásra, ahol az eljárás tartalmaz-za a virnsvektor beadását legalább egy agyksmtába, amely laterális agykamtúk és a »egyedik agykamra alkotta csoportból választott, miáltal a franszgén exprcsszálódik és SMN-I jut a gerittcvelöbe. ti. HüvlN-l-transzgértt tartalmazó, re k táti hinaus AAV-virnsvektor alkalmazása személyben spinális izomatróísa kezelésére szolgáló gyógyszer előállítására, ahol a kezelés tartalmazza a vlmsvektor bejuttatását legalább egy agykamrába, amely laterális agykamrák és a negyedik agykttmra alkotta csoportból választott, miáltal a transzgén expresszálódik.
  6. 12, Az 1. igénypont vagy a KE igénypont szerinti rekombináns virusvektor az ott meghatározott alkalmazásra. ahol a transzgéntermék protein, araeh a HIV 1 \ 1 -proteinjének proleintranszdukciós domenjéhő! 11 antiircisavas .«totivumstJtnrtaltiiaz.
  7. 13. A Π, igénypont szennti alkalmazás, abcs! a transzgéntermék protein, amely a HÍV Ï AT-proteinjének proteintrattszdukeios doménjebot 11 ,uninosa\,ts motívumot tartalmaz;
HUE07795835A 2006-06-07 2007-06-07 Génterápia amiotróf laterális szklerózis és egyéb gerincvelõ-betegségek ellen HUE031156T2 (hu)

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HUE07795835A HUE031156T2 (hu) 2006-06-07 2007-06-07 Génterápia amiotróf laterális szklerózis és egyéb gerincvelõ-betegségek ellen

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