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Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology [CRISPR/Cas9], signaling an innovative advancement in the field of gene therapy.

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Nearly a decade ago, Emmanuelle Charpentier and Jennifer Doudna embarked on a journey that would transform the field of science. Their groundbreaking research on the CRISPR system revolutionized gene editing and ultimately earned them the 2020 Nobel Prize in Chemistry. Watch their captivating short film, offering insights into the process of scientific discovery, collaboration, and groundbreaking experimentation.

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Adeno-associated virus (AAV) is reckoned as one of the most propitious vectors for gene research and therapy. This perception is due to an amalgamation of characteristics such as its variant types, minimal immunogenicity, extensive safety considerations, the extensive range of host cells it can infect (including both dividing and non-dividing cells), its potent dissemination capabilities, and its ability to facilitate prolonged gene expression.

In the context of the Patsnap Bio Sequence Database, it's possible to extract sequences analogous to definite AAV sequences and examine new AAV data from sources accessible to the public, thereby providing a catalyst for advanced AAV research.