(19)
(11)EP 3 540 054 A3

(12)EUROPEAN PATENT APPLICATION

(88)Date of publication A3:
09.10.2019 Bulletin 2019/41

(43)Date of publication A2:
18.09.2019 Bulletin 2019/38

(21)Application number: 19156264.4

(22)Date of filing:  07.06.2007
(51)International Patent Classification (IPC): 
C12N 15/00(2006.01)
A01K 67/027(2006.01)
(84)Designated Contracting States:
AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IS IT LI LT LU LV MC MT NL PL PT RO SE SI SK TR

(30)Priority: 07.06.2006 US 81141906 P

(62)Application number of the earlier application in accordance with Art. 76 EPC:
12152550.5 / 2489733
07795835.3 / 2029742

(71)Applicant: Genzyme Corporation
Cambridge, MA 02142 (US)

(72)Inventors:
  • DODGE, James
    Shrewsbury, MA 01545 (US)
  • SHIHABUDDIN, Lamya
    West Newton MA 02465 (US)
  • O'RIORDAN, Catherine R.
    Waban, MA 02468 (US)

(74)Representative: Rai, Monika et al
Mathys & Squire LLP The Shard 32 London Bridge Street
London SE1 9SG
London SE1 9SG (GB)

  


(54)GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS


(57) This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.





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