U7snRNA-Based Exon Skipping in HCM Mouse Model
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Questions and Answers

What is the purpose of the Terms and Conditions on Wiley Online Library?

  • To outline the production of adeno-associated virus vectors
  • To govern the use of OA articles under the Creative Commons License (correct)
  • To describe the use of morpholino antisense oligonucleotides
  • To provide rules for Duchenne muscular dystrophy research
  • What is the main topic of the 2012 study by Goyenvalle et al.?

  • Production of adeno-associated virus vectors
  • Exon skipping for Duchenne muscular dystrophy (correct)
  • Neonatal gene therapy using AAV-based vectors
  • Gene therapy for hemophilia A
  • What is the purpose of the study by Heemskerk et al. in 2009?

  • To develop a new method for Duchenne muscular dystrophy treatment
  • To study the use of RNA-targeting approaches for neuromuscular diseases
  • To compare AAV vectors for gene therapy
  • To compare 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for exon skipping (correct)
  • What is the title of the 2006 study by Inagaki et al.?

    <p>Robust systemic transduction with AAV vectors in mice</p> Signup and view all the answers

    What is the main topic of the 2003 study by Grimm et al.?

    <p>Production of adeno-associated virus vectors of serotypes 1 to 6</p> Signup and view all the answers

    What is the title of the 2012 study by Hu and Lipshutz?

    <p>AAV-based neonatal gene therapy for hemophilia A</p> Signup and view all the answers

    What is the topic of the study by Le Roy et al. in 2009?

    <p>RNA-targeting approaches for neuromuscular diseases</p> Signup and view all the answers

    What is the purpose of AAV vectors in the study by Inagaki et al.?

    <p>To achieve robust systemic transduction in mice</p> Signup and view all the answers

    What is the topic of the study by Heemskerk et al. in 2009?

    <p>In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides</p> Signup and view all the answers

    What is the main topic of the study by Goyenvalle et al. in 2012?

    <p>Engineering multiple U7snRNA constructs to induce single and multi-exon skipping</p> Signup and view all the answers

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