mucopolysaccharidosis type I
To determine the potential for treatment of mucopolysaccharidosis type I using a gene delivery approach, a recombinant adeno-associated virus (AAV) vector, vTRCA1, transducing the human iduronidase (IDUA) gene was constructed and 1 x 10(10) particles were injected intravenously into 1-day-old Idua(-/-) mice.
To determine the potential for treatment of <span class="disease" id="15194053-2-44-72">mucopolysaccharidosis type I</span> using a gene delivery approach, a recombinant adeno-associated virus (AAV) vector, vTRCA1, transducing the human iduronidase (<span class="gene" id="15194053-2-199-203">IDUA</span>) gene was constructed and 1 x 10(10) particles were injected intravenously into 1-day-old Idua(-/-) mice.