Sökning: "splice-switching oligonucleotide"

Visar resultat 1 - 5 av 6 avhandlingar innehållade orden splice-switching oligonucleotide.

  1. 1. Specificity of antisense oligonucleotide derivatives and cellular delivery by cell-penetrating peptides

    Författare :Peter Guterstam; Ülo Langel; Matthew Wood; Stockholms universitet; []
    Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; cell-penetrating peptide; splice-switching oligonucleotide; oligonucleotide derivative; Neurochemistry; Neurokemi; Neurochemistry and Molecular Neurobiology; neurokemi med molekylär neurobiologi;

    Sammanfattning : Atypical gene expression has a major influence on the disease profile of several severe human disorders. Oligonucleotide (ON) based therapeutics has opened an avenue for compensating deviant protein expression by acting on biologically important nucleic acids, mainly RNAs. LÄS MER

  3. 2. Pharmaceutical insights into improving oligonucleotide delivery and efficacy

    Författare :Osama Saher; Karolinska Institutet; Karolinska Institutet; []
    Nyckelord :;

    Sammanfattning : The power of gene therapy lies in their capacity to target a disease on a genetic level. Nucleic acid containing therapeutics can be utilized to treat, diagnose or prevent a possible disease or condition. However, the successful delivery of such molecules constitutes a major limitation. LÄS MER

  4. 3. Towards smart antisense and antigene therapeutics : design and synthesis of novel oligonucleotide based bioconjugates

    Författare :Martina Jezowska-Herrera; Karolinska Institutet; Karolinska Institutet; []
    Nyckelord :;

    Sammanfattning : The use of nucleic acid technology has been of great importance in life science research. In several clinical trials patients with genetic disease are treated with help of innovations based on oligonucleotide therapy. The use of it is unfortunately limited in efficacy due to e.g. LÄS MER

  5. 4. Optimization of the formulation and design of oligonucleotide-based pharmaceuticals for the purpose of gene therapy

    Författare :Eman M Zaghloul; Karolinska Institutet; Karolinska Institutet; []
    Nyckelord :;

    Sammanfattning : Oligonucleotides (ONs) are short sequences of nucleic acids which may be used in a therapeutic context to modulate gene expression. According to their target, ONs can be classified into two main classes: antisense ONs which target mRNA and antigene ONs that target chromosomal DNA. LÄS MER

  7. 5. Oligonucleotide therapies for RNA and DNA : modulation of natural splice-variants, DNA structure and characterization of new synthetic nucleotides and reporter cell lines

    Författare :Cristina S J Rocha; Karolinska Institutet; Karolinska Institutet; []
    Nyckelord :;

    Sammanfattning : Oligonucleotide therapy is an evolving field having shown fast and important developments in the last years. From genetics to metabolic, inflammatory, immunodeficiency diseases, cancer and viral infections the medical applications for this type of therapy are becoming broader every day. LÄS MER