genetic therapies in sickle cell disease


Hi, I want to help me to make power point in this topic , to have 15 slides ( without the cover page and the references). first I want to talk about sickle cell disease and give idea what it is ( history, diagnosis, lab test , treatments). then talk about the gene therapy in SCD.Genetic Therapies in Sickle Cell Disease
Genomic medicine is rapidly changing and seems to hold enormous promise in the
fight against disease. The field has made tremendous progress in recent years, but
not on the scale that one might expect. Complex diseases are influenced by several
hereditary and environmental factors, as well as an individual’s lifestyle. Taking
genetic-environmental factors into account can aid in the identification of risk
factors linked with a specific disease and lead to individualized targeted therapy.
Sickle cell disease (SCD) is a genetic blood disease with severe and potentially lifethreatening manifestations that affects millions worldwide. It was once assumed to
be a monogenetic disease, but due to the involvement of HbF genes, it is now
regarded as a multigenic complex disease. Currently, the treatment options are
limited. They include pain management and blood transfusions. The only cure for
sickle cell disease now available is bone marrow transplantation from a healthy
donor. Identifying a compatible donor, immunological rejection of the transplant,
and graft-versus-host disease are all key hurdles with this method. Treatments for
SCID, eye problems, HIV, sickle cell anaemia, cystic fibrosis, congestive heart
failure, haemophilia, cancer, and other genetic illnesses are now being evaluated in
gene therapy clinical studies. Gene therapy is a promising treatment for sickle cell
disease. Two general approaches to gene therapy for SCD are gene addition and
gene editing. In gene addition, the genes are transferred into the genome via a
vector. In gene editing, permanent genomic changes are made, which involves the
removal or replacement of DNA sequences.
Successful clinical trials are ongoing for CRISPR-CAS9 gene therapy as a
promising treatment for sickle cell disease. To analyse this, a primary data paper on
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and B-Thalassemia was used.
BCL11A is a transcription factor in erythroid cells that suppresses the expression of
γ -globin and foetal haemoglobin. With CRISPR-Cas9 targeting the BCL11A
erythroid-specific enhancer, CD34+ hematopoietic stem and progenitor cells were
obtained from healthy donors. Almost 80% of the alleles at this locus were
modified, with no signs of off-target editing. CTX001 was manufactured from these
CD34+ cells by editing with CRISPR-Cas9 with the use of a single-guide RNA
molecule. After myeloablation, the patients received CTX001. More than a year
later, both patients had high levels of allelic editing in bone marrow and blood,
increases in foetal haemoglobin that were distributed pan-cellularly, transfusion
independence, and the elimination of vaso-occlusive episodes. At month 15, the
foetal haemoglobin level rose to 43.2% and the sickle haemoglobin increased to
52.3%. F-cell expression was 99.9% at month 5 and was maintained at nearly 100%
through month 15.
Despite the progress, gene therapy’s long-term efficacy and safety are still being
established. Due to challenges such as technological difficulties in therapy, ethical
concerns, and financial considerations, it has had only limited success.Advances in
technology and study have contributed to a greater understanding and ability to read
the genome, but the complexity of diseases and the ambiguity surrounding gene
therapy have slowed medical development.

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