Advanced Cell Bio

CRISPR

Clustered Regularly Interspaced Short Palindromic Repeats

Advantage of CRISPR-Cas9

Precise and efficient gene editing

Difference of CRISPR-Cas9

More precise and easier to use than previous gene editing techniques

Role of gRNA in CRISPR-Cas9

Guides Cas9 to the target DNA sequence

Original function of CRISPR-Cas system

Bacterial immune system against viruses

Diseases CRISPR-Cas9 used for in clinical trials

Sickle cell anemia, beta-thalassemia, and Leber congenital amaurosis

PAM in CRISPR-Cas9 system

Protospacer Adjacent Motif, necessary for Cas9 to bind and cut DNA

Enzymes in Cas protein complex

Cas9 and Cas1-Cas2

Cas9 cuts DNA how many bases upstream

Three bases upstream from the binding sequence

Repair mechanism creating mutation

Non-homologous end joining (NHEJ)

CRISPR-Cas9 system in bacteria akin to

Adaptive immune system

Frequency of 'GG' PAM sequences in mammalian genome

Approximately 18,750

Advantages of nucleic acid-based vaccines

Rapid development and potential for broad protection

Key advantage of mRNA vaccines over DNA vaccines

No risk of integration into the host genome

Drawback of viral vector-based vaccines

Risk of pre-existing immunity and potential for insertional mutagenesis

Early milestone in mRNA vaccine development

Demonstration of protein expression in vivo

Key development in production of mRNA

Introduction of modified nucleoside pseudouridine

Early advancement in nucleic acid delivery

Lipid nanoparticles for efficient cellular uptake

Contribution of Katalin Karikó

Pioneering mRNA research and development

Drew Weissman's background and expertise

Immunologist specializing in RNA biology

Key finding from Karikó and Weissman collaboration

Modified nucleosides reduce immune response to mRNA

Finding on activation of dendritic cells by mRNA

Induction of strong immune response

Focus of main companies in mRNA technology

Vaccine development, cancer immunotherapy, and protein replacement therapy

Aspect of mRNA-based vaccine against MERS-CoV

Rapid response to emerging infectious diseases

Future directions in mRNA vaccine technology

Personalized vaccines, combination therapies, and broader vaccine applications