The role of CRISPR in the treatment of sickle cell anemia

dc.contributor.advisorSzentesiné Szirák, Krisztina
dc.contributor.advisordeptHumángenetikai Tanszékhu_HU
dc.contributor.authorNwokorie, Ugochukwu Uzoma
dc.contributor.departmentDE--Általános Orvostudományi Karhu_HU
dc.contributor.opponentPolgár, Zsuzsanna
dc.contributor.opponentJambrovics, Károly
dc.contributor.opponentdeptDebreceni Egyetem::Általános Orvostudományi Kar::Orvosi Vegytani Intézethu_HU
dc.contributor.opponentdeptDebreceni Egyetem::Általános Orvostudományi Kar::Biokémiai és Molekuláris Biológiai Intézethu_HU
dc.date.accessioned2021-09-16T07:45:49Z
dc.date.available2021-09-16T07:45:49Z
dc.date.created2021-09-03
dc.description.abstractSickle cell anemia (SCA) is an autosomal recessive disease that gives rise to abnormal hemoglobin, thus impairing the oxygen carrying ability of the red blood cells (RBCs).Up till this point, SCA treatment has been mostly pharmaceutical accompanied by lifestyle modifications. However, since the advent of more precise genome editing tools like CRISPR, there are new, more promising possibilities for SCA therapy.CRISPR-Cas9 genome editing is a technique, based on a bacterial defense system, that can be adapted to modify genes of living organisms. With this tool, a programmed complex consisting of Cas9 nuclease coupled with a single-guide RNA (sgRNA) is delivered into a cell using a vector, and this complex then cuts the genome at a specified location, giving certain desired outcomes based on the induced mutation.hu_HU
dc.description.courseáltalános orvoshu_HU
dc.description.courselangangolhu_HU
dc.description.degreeegységes, osztatlanhu_HU
dc.format.extent43hu_HU
dc.identifier.urihttp://hdl.handle.net/2437/321752
dc.language.isoenhu_HU
dc.subjectCRISPRhu_HU
dc.subjectsickle cell anemiahu_HU
dc.subjectMendelian diseasehu_HU
dc.subjectgene editinghu_HU
dc.subjectCRISPR-Cas9hu_HU
dc.subjectgenome editinghu_HU
dc.subject.dspaceDEENK Témalista::Biológiai tudományok::Humángenetikahu_HU
dc.titleThe role of CRISPR in the treatment of sickle cell anemiahu_HU
dc.title.translatedA CRISPR szerepe a sarlósejtes vérszegénység kezelésébenhu_HU
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