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Human Genome Editing
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debbie394
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What does the following mean?
Can you give me a summary and tell me what induced molecular remission is?
The team at Great Ormond Street Hospital (GOSH) used modified T-cells from donors, known as UCART19 cells, to treat a one-year-old child with an aggressive form of acute lymphoblastic leukaemia (ALL) who had already had unsuccessful chemotherapy and for whom palliative care was the only other remaining option. The treatment worked by editing healthy donor T-cells, using molecular tools (TALENs) to cut specific genes in order to make them behave in two ways. Firstly, they become invisible to a powerful leukaemia drug, Alemtuzumab, that would usually kill them and, secondly, they are reprogrammed specifically to target and fight against leukaemia cells. The team at GOSH and the Institute of Child Health, with investigators at University College London and the biotech company Cellectis, had been developing ‘off-the-shelf’ banks of these donor T-cells, the first of which was due to be used for final stage testing ahead of clinical trials. However, the team received a request for therapy on a compassionate basis for an 11-month old girl with refractory relapsed B-acute lymphoblastic leukaemia, and were able to provided treatment under UK special therapy regulations. At an early stage of follow up, the team reports induced molecular remission in this patient where all other treatments had proved ineffective.
Can you give me a summary and tell me what induced molecular remission is?
The team at Great Ormond Street Hospital (GOSH) used modified T-cells from donors, known as UCART19 cells, to treat a one-year-old child with an aggressive form of acute lymphoblastic leukaemia (ALL) who had already had unsuccessful chemotherapy and for whom palliative care was the only other remaining option. The treatment worked by editing healthy donor T-cells, using molecular tools (TALENs) to cut specific genes in order to make them behave in two ways. Firstly, they become invisible to a powerful leukaemia drug, Alemtuzumab, that would usually kill them and, secondly, they are reprogrammed specifically to target and fight against leukaemia cells. The team at GOSH and the Institute of Child Health, with investigators at University College London and the biotech company Cellectis, had been developing ‘off-the-shelf’ banks of these donor T-cells, the first of which was due to be used for final stage testing ahead of clinical trials. However, the team received a request for therapy on a compassionate basis for an 11-month old girl with refractory relapsed B-acute lymphoblastic leukaemia, and were able to provided treatment under UK special therapy regulations. At an early stage of follow up, the team reports induced molecular remission in this patient where all other treatments had proved ineffective.
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