Lecture

Approaches in Gene Therapy

Related lectures (32)

Explores gene therapy for CNS and sensory organs, focusing on rare genetic diseases and advancements in precision medicine.

Production of Viral Vectors

Explores the production of viral vectors for gene therapy applications, emphasizing AAV vectors and the process of large-scale production and purification.

Preclinical and Clinical Trials in SMA Gene Therapy

Explores preclinical and clinical trials in SMA gene therapy, including efficiency in patients, trial results, adverse effects, cellular targeting, and future perspectives.

Types of Viral Vectors

Explores the properties of commonly used viral vectors for gene therapy.

Gene Therapy: Targets and Therapeutics

Explores gene therapy targets, RNA-based therapeutics, and recent advancements in genetic treatments for monogenic diseases and cancer.

Choosing a Viral Vector and Delivery Method

Discusses viral vector selection for gene therapy, with a focus on AAV vectors for SMA1 therapy and CNS delivery.

Non Viral Vectors

Explores the use of non-viral vectors for precise nucleic acid delivery and their limitations in achieving long-term gene expression.

Gene Therapy: Neurological Diseases

Explores gene therapy for neurological diseases, including monogenic and multifactorial disorders, and the use of Cas9 and repair mechanisms.

Gene Therapy: RNA Therapeutics & Monogenic Diseases

Explores gene therapy, RNA therapeutics, monogenic diseases, CAR T cell therapy, and CRISPR-based treatments.

Personalized Medicine: Gene Therapy and CRISPR

Explores personalized medicine, gene therapy, CRISPR technology, gene editing strategies, and ethical implications of synthetic genomics.

Synthetic Biology: Therapeutic Advancement

Introduces synthetic biology for disease treatment, covering RNA therapeutics, drug discovery, and gene circuits.

Alternative Strategies for SMA Gene Therapy

Explores alternative strategies for SMA gene therapy, including antisense oligonucleotides and genome editing.

Comparing Covid-19 Vaccines

Compares Covid-19 vaccines, explains mRNA vaccine mechanism, and discusses virus structure and soap fabrication.

Genome Editing: CRISPR-Cas Applications

Explores the applications of CRISPR-Cas in genome editing, focusing on engineering bacterial genomes, curing genetic diseases, guide RNA simplicity, Cas9 specificity, DNA damage mechanisms, and base editing.

RNA-based Approaches to Disease Treatment

Explores RNA-based disease treatment, synthetic biology impact on therapeutics, and programmable RNA-targeting tools.

Synthetic Biology: Therapeutic Advancement

Delves into cutting-edge tools for disease treatment through synthetic biology.

CRISPR-Based Tools & Diagnostics

Explores CRISPR-based tools and diagnostics for faster, simpler, and cheaper testing methods with high specificity and sensitivity.

AAV Vectors: Development and Applications

Explores the development and applications of AAV vectors, highlighting their specificity and stability for gene therapy.

Genetic Compensation Mechanisms

Delves into genetic compensation mechanisms triggered by mutant mRNA degradation and their implications on human genetics.

Gene Editing and DNA Origami: Innovations in Biotechnology

Discusses recombinant DNA technology, CRISPR/Cas9 gene editing, and DNA origami applications in biotechnology.