Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution
Hemoglobin (Hb) disorders or hemoglobinopathies are groups of blood conditions involving inherited genetic diseases – mostly as single-gene autosomal recessive – that lead to the formation of abnormal Hb structure or inadequate to no production of globin chains in Hbs. Disorders of Hb are a global c...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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Wolters Kluwer Medknow Publications
2020-01-01
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| Series: | Journal of Nature and Science of Medicine |
| Subjects: | |
| Online Access: | http://www.jnsmonline.org/article.asp?issn=2589-627X;year=2020;volume=3;issue=3;spage=146;epage=154;aulast=Al |
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| author | Bader Al Alwan Arwa A Alsubait Bahauddeen M Alrfaei |
| author_facet | Bader Al Alwan Arwa A Alsubait Bahauddeen M Alrfaei |
| author_sort | Bader Al Alwan |
| collection | DOAJ |
| description | Hemoglobin (Hb) disorders or hemoglobinopathies are groups of blood conditions involving inherited genetic diseases – mostly as single-gene autosomal recessive – that lead to the formation of abnormal Hb structure or inadequate to no production of globin chains in Hbs. Disorders of Hb are a global concern since these diseases can cause severe morbidity and early mortality of the affected populations. Treatments vary between chemicals and molecular approaches. The most promising approach is Hb correction. However, the stability of the correction faces a big challenge along with safety concerns. It is worth noting that most of the inherited hemoglobinopathies share common clinical presentations and laboratory findings, although some have distinct features. Hemoglobinopathies with emphasis on recent advances in gene therapy targeting sickle cell disease and thalassemia are discussed in this review. |
| first_indexed | 2024-12-13T08:36:17Z |
| format | Article |
| id | doaj.art-765826614745406fab2bf7b7f6b8adb2 |
| institution | Directory Open Access Journal |
| issn | 2589-627X 2589-6288 |
| language | English |
| last_indexed | 2024-12-13T08:36:17Z |
| publishDate | 2020-01-01 |
| publisher | Wolters Kluwer Medknow Publications |
| record_format | Article |
| series | Journal of Nature and Science of Medicine |
| spelling | doaj.art-765826614745406fab2bf7b7f6b8adb22022-12-21T23:53:39ZengWolters Kluwer Medknow PublicationsJournal of Nature and Science of Medicine2589-627X2589-62882020-01-013314615410.4103/JNSM.JNSM_59_19Hemoglobinopathy correction with CRISPR or not; gene therapy is the solutionBader Al AlwanArwa A AlsubaitBahauddeen M AlrfaeiHemoglobin (Hb) disorders or hemoglobinopathies are groups of blood conditions involving inherited genetic diseases – mostly as single-gene autosomal recessive – that lead to the formation of abnormal Hb structure or inadequate to no production of globin chains in Hbs. Disorders of Hb are a global concern since these diseases can cause severe morbidity and early mortality of the affected populations. Treatments vary between chemicals and molecular approaches. The most promising approach is Hb correction. However, the stability of the correction faces a big challenge along with safety concerns. It is worth noting that most of the inherited hemoglobinopathies share common clinical presentations and laboratory findings, although some have distinct features. Hemoglobinopathies with emphasis on recent advances in gene therapy targeting sickle cell disease and thalassemia are discussed in this review.http://www.jnsmonline.org/article.asp?issn=2589-627X;year=2020;volume=3;issue=3;spage=146;epage=154;aulast=Alcrisprhemoglobin disordersthalassemia |
| spellingShingle | Bader Al Alwan Arwa A Alsubait Bahauddeen M Alrfaei Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution Journal of Nature and Science of Medicine crispr hemoglobin disorders thalassemia |
| title | Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution |
| title_full | Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution |
| title_fullStr | Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution |
| title_full_unstemmed | Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution |
| title_short | Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution |
| title_sort | hemoglobinopathy correction with crispr or not gene therapy is the solution |
| topic | crispr hemoglobin disorders thalassemia |
| url | http://www.jnsmonline.org/article.asp?issn=2589-627X;year=2020;volume=3;issue=3;spage=146;epage=154;aulast=Al |
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