Adeno-associated virus-mediated delivery of GDNF to skeletal muscles
AssigneeGenzyme Corporation
InventorsLijun Wang, Shin-ichi Muramatsu, Imaharu Nakano, Hiroaki Mizukami, Keiya Ozawa
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Abstract
Compositions and methods for delivering GDNF to skeletal muscles to result in a therapeutic effect are disclosed. The compositions and methods use adeno-associated virus (AAV)-based gene delivery systems. The methods are useful for treating motoneuron diseases, such as amyotrophic lateral sclerosis (ALS).
Claims7
A method of delivering a recombinant adeno-associated virus (AAV) virion to a muscle cell or muscle tissue of a mammalian subject with amyotrophic lateral sclerosis, said method comprising:
- (a) providing a recombinant AAV virion which comprises a polynucleotide encoding a glial cell line-derived neurotrophic factor (GDNF) operably linked to control elements capable of directing the in vivo transcription and translation of said GDNF;
- and
- (b) delivering said recombinant AAV virion directly into said muscle cell or muscle tissue of said subject, whereby said GDNF is expressed at a level which provides a therapeutic effect in said mammalian subject.
A method of delivering a recombinant adeno-associated virus (AAV) virion to a skeletal muscle of a human subject with amyotrophic lateral sclerosis, said method comprising:
- (a) providing a recombinant AAV virion, said recombinant AAV virion comprising a polynucleotide encoding a human glial cell line-derived neurotrophic factor (GDNF) operably linked to control elements capable of directing the in vivo transcription and translation of said GDNF;
- and
- (b) delivering said recombinant AAV virion directly into skeletal muscle of said subject in vivo, whereby said GDNF is expressed at a level which provides a therapeutic effect in said human subject.
A method of treating a mammalian subject with amyotrophic lateral sclerosis, said method comprising administering into skeletal muscle of said subject a composition comprising recombinant adeno-associated virus (AAV) virions that comprise a polynucleotide encoding a glial cell line-derived neurotrophic factor (GDNF) polypeptide operably linked to expression control elements capable of directing the in vivo transcription and translation of said GDNF, to provide a therapeutic effect.
Dependencies are inferred from the claim text.