Ghazala Nathu*, Saahil Patel and Adila Nathu


Recent advances in managing Sickle Cell Disease (SCD) improved the survival of pediatric patients and the quality of life in adult patients significantly. Disease-modifying drug therapies hydroxyurea, voxelotor, crizanlizumab, and L-glutamine reduced pain crises and severe complications. Allogeneic hematopoietic stem cell transplantation using sibling donors is currently the only standard curative option. However, the matched-related donor is available for a smaller proportion of patients. Cord blood transplantation and haploidentical transplantation with a modified conditioning regiment are expanding the allogeneic donor pool making the therapy available to most patients. In vitro expanded cord blood cells are effectively used in adult patients. However, the most promising treatment for a cure is gene therapy. Several gene therapy methodologies have been successful in recent clinical trials. A new non-sickling allele can be introduced to hematopoietic stem cells by lentiviral vectors. The sickle allele could be silenced while enhancing other hemoglobin alleles, most notably HgbF. The sickle allele could be gene-edited through homologous-directed repair and CRISPR technology. Editing regulatory sequences boost non-sickle hemoglobin production. Multidisciplinary expertise and effort are combined to standardize metrics and compare different therapeutic options. The Cure Sickle Cell Initiative supports research development encompassing pharmacologic, biotherapies, and genetic advancements. The workgroups are bringing together basic and translational researchers, clinicians, and patients through education and motivation in a joint effort to support high-quality clinical trials toward curative therapies.

Keywords: .

[Full Text Article]


Forgot Password  |  Register


Best Paper Awards

European Journal of Biomedical and Pharmaceutical Sciences (EJBPS) will give best paper award in every issue in the form of money along with certificate to promote research activity of scholar.

Best Article of current issue :

Dr. Dhrubo Jyoti Sen

Download Article : Click here

News & Updation


    MARCH 2023 Issue has been successfully launched on 1 MARCH 2023.

  • EJBPS New Impact Factor

    Its our Pleasure to Inform you that EJBPS Impact Factor has been increased from 5.467 to 6.044, due to high quality Publication at International Level.

  • Index Copernicus Value

    EJBPS Received Index Copernicus Value 77.3, due to High Quality Publication in EJBPS at International Level

  • Journal web site support Internet Explorer, Google Chrome, Mozilla Firefox, Opera, Saffari for easy download of article without any trouble.


  • Article Invited for Publication

    Dear Researcher, Article Invited for Publication  in EJBPS coming Issue.


UG/PG/Ph.D Research Publication

Research Scholar of UG/PG/Ph.D can Submit their Research Article/Review Article/Case Study/Short Communication for Publication in EJBPS


Copyright From

Covering Letter

                        Author Instruction