1. Volume 11, Issue 1, Pages 48-56 (January 2005)
Replacing acid α-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers 
Nina Raben, Tokiko Fukuda, Abigail L. Gilbert, Deborah de Jong, Beth L. Thurberg, Robert J. Mattaliano, Peter Meikle, John J. Hopwood, Kunio Nagashima, Kanneboyina Nagaraju, Paul H. Plotz 
Molecular Therapy 
Volume 11, Issue 1, Pages (January 2005)
DOI: /j.ymthe
Copyright © 2004 The American Society of Gene Therapy Terms and Conditions

2. Fig. 1
Reduction in lysosomal glycogen storage in different muscle groups after ERT with rhGAA (20 mg/kg/biweekly). Complete glycogen clearance was observed in soleus muscle after 12 injections: (A) untreated age-matched control; (B) treated. No significant clearance was detected in quadriceps even after 24 injections: (C) untreated age-matched control; (D) treated. (E) Histologic glycogen (as shown in A through D, using the 40× objective) was quantified for soleus, plantaris, gastrocnemius, and quadriceps using the histomorphometric program MetaMorph. Glycogen is expressed as the percentage of tissue area occupied by glycogen.
Molecular Therapy  , 48-56DOI: ( /j.ymthe )
Copyright © 2004 The American Society of Gene Therapy Terms and Conditions

3. Fig. 2
(A) PAS-stained section of gastrocnemius muscle showing significant amounts of residual glycogen. (B) Western blot immunodetection of GAA protein in treated mouse (shown in A) and wild-type mouse. H, heart; G, gastrocnemius; S, soleus. Note that in this particular animal GAA protein is more abundant in gastrocnemius then in soleus. Immunodetection of vinculin was used for control loading. (C) Western blot immunodetection of GAA protein in skeletal muscle biopsies from control wild-type mouse (lane 1), human quadriceps from an unaffected individual (lane 2), and treated tGaa−/− mouse (lanes 3 and 4). Q, quadriceps; P, plantaris. Each lane was loaded with 50 μg of protein. The samples in lanes 1 and 2 had equivalent enzymatic activity. Two smaller (∼20/25 kDa) products were also observed on Western blots (not shown).
Molecular Therapy  , 48-56DOI: ( /j.ymthe )
Copyright © 2004 The American Society of Gene Therapy Terms and Conditions

4. Fig. 3
Somatic induction of the GAA transgene expression in the liver of the knockout mice. The GAA transgene was induced in a 3-week-old mouse by dox removal; the tissues were analyzed (A and B) 3 weeks or (C and D) 5 weeks after dox was removed. PAS-stained sections of (A and C) cardiac muscle and (B and D) soleus muscle show significant metabolic cross-correction in the heart and partial correction in some of the fibers of soleus muscle after a 5-week washout period.
Molecular Therapy  , 48-56DOI: ( /j.ymthe )
Copyright © 2004 The American Society of Gene Therapy Terms and Conditions

5. Fig. 4
Somatic induction of the GAA transgene in mice from the 6 weeks gene-off/19 weeks gene-on group. Areas of PAS-stained sections with a significant amount of residual glycogen in (A) gastrocnemius and (B) quadriceps are shown.
Molecular Therapy  , 48-56DOI: ( /j.ymthe )
Copyright © 2004 The American Society of Gene Therapy Terms and Conditions

6. Fig. 5
Western blot immunodetection of (A) lysosomal/endosomal markers and (B) proteins involved in clathrin-mediated endocytosis.
Molecular Therapy  , 48-56DOI: ( /j.ymthe )
Copyright © 2004 The American Society of Gene Therapy Terms and Conditions

7. Fig. 6
Electron micrographs of skeletal muscle biopsy from a 4-month-old tGaa−/− mouse. (A) Accumulation of single-membrane-bound intralysosomal glycogen in soleus (type I) muscle (asterisk). (B and C) Accumulation of intralysosomal glycogen and multiple small and large coalesced autophagic vacuoles in gastrocnemius (type II) muscle (arrows).
Molecular Therapy  , 48-56DOI: ( /j.ymthe )
Copyright © 2004 The American Society of Gene Therapy Terms and Conditions