Sökning: "nucleic acid delivery"

Visar resultat 21 - 25 av 35 avhandlingar innehållade orden nucleic acid delivery.

  1. 21. 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. 22. Profiling and exploiting lipid-based nanoparticles in vitro and in vivo

    Författare :Helena Sork; Karolinska Institutet; Karolinska Institutet; []
    Nyckelord :;

    Sammanfattning : One of the major hurdles for therapeutic applications is the efficient delivery of bioactive molecules to the site of action. The high flexibility and biosafety of lipid-based nanoparticles has greatly enhanced their employment as delivery systems not only for synthetic but also for natural molecules such as proteins and nucleic acids. LÄS MER

  4. 23. Cell-penetrating peptides; chemical modification, mechanism of uptake and formulation development

    Författare :Kariem Ezzat; Ülo Langel; Steven Dowdy; Stockholms universitet; []
    Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; cell penetrating peptides; gene therapy; scavenger receptors; pharmaceutical formulation; solid dispersion; neurokemi med molekylär neurobiologi; Neurochemistry with Molecular Neurobiology;

    Sammanfattning : Gene therapy holds the promise of revolutionizing the way we treat diseases. By using recombinant DNA and oligonucleotides (ONs), gene functions can be restored, altered or silenced according to the therapeutic need. LÄS MER

  5. 24. CELL PENETRATING PEPTIDES : CHEMICAL MODIFICATION AND FORMULATION DEVELOPMENT

    Författare :Kariem Ezzat; Ülo Langel; Astrid Gräslund; Stockholms universitet; []
    Nyckelord :NATURAL SCIENCES; NATURVETENSKAP; Neurochemistry and Molecular Neurobiology; neurokemi med molekylär neurobiologi;

    Sammanfattning : Cell penetrating peptides (CPPs) have been extensively studied and exploited as drug delivery vectors for a wide variety of therapeu-tic cargos. However, several issues remain to be addressed regarding the enhancement of their efficiency and stability. LÄS MER

  7. 25. 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