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Aav2/9 cmv LARGE1

Intervention

1 paper4 findings

C, W, M, H: cells, worms or flies, mice, humans. Dark: independent labs found the same change there. Light: one lab or one study. Outline: nothing yet.

  • 1 lab in 1 country
  • Funders: National Institute of Diabetes and Digestive and Kidney Diseases and 1 more
  • Animals1

Outcomes

4

Matriglycan

upin mice1

1 study

  1. Aav2/9 cmv LARGE1 increases matriglycan in mice.

    “restores matriglycan expression on dystroglycan”

    Animalsin older myd mice with severe muscular dystrophy

    Large1 gene transfer in older myd mice with severe muscular dystrophy restores muscle function and greatly improves survival

    Science advances25 May 2022

More on Matriglycan

Skeletal muscle pathophysiology

downin mice1

1 study

  1. Aav2/9 cmv LARGE1 decreases skeletal muscle pathophysiology in mice.

    “attenuates skeletal muscle pathophysiology”

    Animalsin older myd mice with severe muscular dystrophy

    Large1 gene transfer in older myd mice with severe muscular dystrophy restores muscle function and greatly improves survival

    Science advances25 May 2022

Motor and respiratory function

upin mice1

1 study

  1. Aav2/9 cmv LARGE1 improves motor and respiratory function in mice.

    “improves motor and respiratory function”

    Animalsin older myd mice with severe muscular dystrophy

    Large1 gene transfer in older myd mice with severe muscular dystrophy restores muscle function and greatly improves survival

    Science advances25 May 2022

Survival

upin mice1

1 study

  1. Aav2/9 cmv LARGE1 extends lifespan survival in mice.

    “which collectively and markedly extends survival.”

    Animalsin older myd mice with severe muscular dystrophy

    Large1 gene transfer in older myd mice with severe muscular dystrophy restores muscle function and greatly improves survival

    Science advances25 May 2022

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