Scientific rationale for the development of gene therapy strategies for Parkinson's disease.

Forskningsoutput: TidskriftsbidragArtikel i vetenskaplig tidskrift


The ever-evolving understanding of the neuronal systems involved in Parkinson's disease together with the recent advances in recombinant viral vector technology has led to the development of several gene therapy applications that are now entering into clinical testing phase. To date, four fundamentally different approaches have been pursued utilizing recombinant adeno-associated virus and lentiviruses as vectors for delivery. These strategies aim either to restore the lost brain functions by substitution of enzymes critical for synthesis of neurotransmitters or neurotrophic factors as a means to boost the function of remaining neurons in the diseased brain. In this review we discuss the differences in mechanism of action and describe the scientific rationale behind the currently tested gene therapy approaches for Parkinson's disease in some detail and pinpoint their individual unique strengths and weaknesses.


Enheter & grupper

Ämnesklassifikation (UKÄ) – OBLIGATORISK

  • Neurovetenskaper
Sidor (från-till)703-713
TidskriftBiochimica et Biophysica Acta - Molecular Basis of Disease
Utgåva nummer7
StatusPublished - 2009
Peer review utfördJa


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