Search for dissertations about: "splice correction"

Showing result 1 - 5 of 7 swedish dissertations containing the words splice correction.

  1. 1. Vectorization of oligonucleotides with cell-penetrating peptides : Characterization of uptake mechanisms and cytotoxicity

    Author : Samir EL Andaloussi; Ülo Langel; Alain Prochiantz; Stockholms universitet; []
    Keywords : MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; CPP; endocytosis; splice correction; siRNA; PNA; cargo delivery; M918; Neurochemistry; Neurokemi; Neurochemistry and Molecular Neurobiology; neurokemi med molekylär neurobiologi;

    Abstract : The hydrophobic plasma membrane constitutes an indispensable barrier for cells in living animals. Albeit being pivotal for the maintenance of cells, the inability to cross the plasma membrane is still one of the major obstacles to overcome in order to progress current drug development. READ MORE

  2. 2. Design and application of RNA therapeutics for splice site mutations

    Author : Burcu Bestas; Karolinska Institutet; Karolinska Institutet; []
    Keywords : MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP;

    Abstract : Precursor messenger RNA splicing is one of the most fundamental and complex mechanisms in eukaryotes. Over 90% of the human genes undergo alternative splicing, which is essential for the regulation of gene expression. A dynamic RNA-protein complex called the spliceosome catalyzes splicing. READ MORE

  3. 3. Cell-penetrating peptides in protein mimicry and oligonucleotide delivery : Applications and mechanisms

    Author : Henrik Johansson; Ülo Langel; Michael Gait; Stockholms universitet; []
    Keywords : MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; peptide; oligonucleotide; cell-penetrating peptide; PNA; splicing; Neurochemistry; Neurokemi; Neurochemistry and Molecular Neurobiology; neurokemi med molekylär neurobiologi;

    Abstract : The plasma membrane functions as a barrier, restricting entry of hydrophilic pharmaceutical agents. Cell-penetrating peptides (CPPs) are capable of transporting bioactive cargos into the cell and have consequently been extensively investigated for their mechanism of entry and capability to deliver various cargos spanning from peptides to plasmids. READ MORE

  4. 4. Delivery of gene-regulating agents: Internalization mechanisms and novel vectors

    Author : Per Lundin; Karolinska Institutet; Karolinska Institutet; []
    Keywords : MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP;

    Abstract : The sequencing of the human genome was expected to generate a veritable explosion of therapeutics for regulation of gene function, either through modulation of gene expression or through the replacement of defect genes. However, nucleic acid-based pharmacological agents suffer from issues of low bioavailability and unfavorable pharmacokinetics, wherefore these prospects have not yet been realized. READ MORE

  5. 5. Nucleic acid delivery: reports from the search of the Magic Bullet

    Author : Joana Viola; Karolinska Institutet; Karolinska Institutet; []
    Keywords : MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP;

    Abstract : Gene therapy is regarded as one of the most promising therapeutic approaches, since it has the potential to treat disorders by correcting malformations at the nucleic acids level rather than proteins, as opposed to conventional medicine. However, for non-viral gene therapy to successfully fulfill the requirements associated to the “magic bullet” has proven difficult. READ MORE