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Cell and Gene Therapy

Cell and Gene Therapy (CGT) and its applications.

Cell and Gene Therapy Overview

Cell and gene therapy are innovative medical approaches that involve using living cells or modifying genes to treat diseases. Cell therapy uses cells to restore damaged tissues, while gene therapy modifies genes to correct genetic disorders. These therapies offer targeted and personalised treatments with prospects of long-lasting effects and have potential to transform the lives of countless individuals and improve the overall health and well-being of society.

Cell and Gene Therapy Overview

Researchers across various scientific and medical fields use cell and gene therapy in their work. Geneticists and molecular biologists are involved in developing gene therapy strategies, designing vectors to deliver therapeutic genes, and exploring gene editing techniques like CRISPR-Cas9. Immunologists focus on understanding the immune system's response to cell and gene therapies and perfecting immune cell-based treatments. Oncologists investigate the application of cell and gene therapies in cancer treatment, including personalised cancer vaccines and engineered immune cells. In regenerative medicine, cell therapy aims to restore damaged tissues, while immunotherapies enhance the immune system's ability to fight diseases. Additionally, cell and gene therapies provide hope for rare genetic diseases and hold potential for treating neurological disorders by replacing damaged neurons or supplying neuroprotective effects.


CAR-T cell therapy is an innovative immunotherapy that involves genetically modifying a patient's own T cells to express chimeric antigen receptors (CARs), enabling them to recognise and attack cancer cells. This therapy has shown remarkable success in treating certain haematological malignancies, such as acute lymphoblastic leukaemia and diffuse large B-cell lymphoma. Ongoing research aims to expand its application to other cancers and explore combination therapies, optimise CAR design, improve manufacturing processes, and mitigate adverse effects. Overall, CAR-T cell therapy holds tremendous promise for personalised cancer treatment and continues to advance through scientific investigations and development. For more information on CAR-T cell therapy, please look here.

Adeno-Associated Viruses

AAV Adeno-Associated Virus diagram
AAV (Adeno-Associated Virus) is a versatile gene therapy vector widely used in the field of life sciences. It can be obtained from various life science distributors as pre-packaged AAV vectors with different serotypes and promoter options, along with helper plasmids for production. AAV-based gene therapy has diverse applications, including targeted gene delivery, gene editing, and gene replacement strategies. It has shown promising results in treating genetic disorders, such as 
inherited retinal diseases and spinal muscular atrophy. Ongoing research focuses on optimising AAV vector design, enhancing transduction efficiency, developing tissue-specific promoters, and addressing immunological challenges for broader clinical applications. AAV-based therapies have the potential to revolutionise the treatment of genetic diseases and hold great promise for the future of precision medicine. For more information on Adeno-Associated Viruses please look here.

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a groundbreaking gene editing technology that has revolutionised the field of life sciences. CRISPR enables precise genome editing by targeting specific DNA sequences, allowing for gene knockout, gene insertion, and gene modification. This technology has numerous applications, ranging from basic research to potential therapeutic interventions. Ongoing research in CRISPR aims to enhance its efficiency, reduce off-target effects, develop new CRISPR systems (such as base editors), and explore ethical and regulatory considerations. CRISPR has transformed the way scientists' study and manipulate genomes, providing unprecedented opportunities for scientific advancements and medical breakthroughs. For more information on CRISPR, please take a look here.

2BScientific not only prides itself on its services, but in doing so we ensure that our suppliers are something to be proud of also! That’s why we have partnered with ACRO Biosystems, Arigo Bio, Cellecta, Cell Biolabs, and Progen to ensure we can provide only the most cutting edge, reliable and accurate reagents. These suppliers provide a range of cell and gene therapy reagents such as vectors, media, recombinant options, high quality AAV reagents and CRISPR libraries.

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