Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders

A new approach is adopted to treat primary immunodeficiency disorders, such as the severe combined immunodeficiency (SCID; e.g., adenosine deaminase SCID [ADA-SCID] and IL-2 receptor X-linked severe combined immunodeficiency [SCID-X1]). The success, along with the feasibility of gene therapy, is und...

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Autores principales:	Odiba Arome Solomon, Okoro Nkwachukwu Oziamara, Durojaye Olanrewaju Ayodeji, Wu Yanjun
Formato:	article
Lenguaje:	EN
Publicado:	De Gruyter 2021
Materias:	clinical trials gene therapy hemoglobin hemophilia b neurodegenerative ocular Biology (General) QH301-705.5
Acceso en línea:	https://doaj.org/article/375714475dc643baa2b39f505edeb293
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spelling	oai:doaj.org-article:375714475dc643baa2b39f505edeb2932021-12-05T14:10:41ZGene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders2391-541210.1515/biol-2021-0033https://doaj.org/article/375714475dc643baa2b39f505edeb2932021-05-01T00:00:00Zhttps://doi.org/10.1515/biol-2021-0033https://doaj.org/toc/2391-5412A new approach is adopted to treat primary immunodeficiency disorders, such as the severe combined immunodeficiency (SCID; e.g., adenosine deaminase SCID [ADA-SCID] and IL-2 receptor X-linked severe combined immunodeficiency [SCID-X1]). The success, along with the feasibility of gene therapy, is undeniable when considering the benefits recorded for patients with different classes of diseases or disorders needing treatment, including SCID-X1 and ADA-SCID, within the last two decades. β-Thalassemia and sickle cell anemia are two prominent monogenic blood hemoglobin disorders for which a solution has been sought using gene therapy. For instance, transduced autologous CD34+ HSCs via a self-inactivating (SIN)-Lentivirus (LV) coding for a functional copy of the β-globin gene has become a feasible procedure. adeno-associated virus (AAV) vectors have found application in ocular gene transfer in retinal disease gene therapy (e.g., Leber’s congenital amaurosis type 2), where no prior treatment existed. In neurodegenerative disorders, successes are now reported for cases involving metachromatic leukodystrophy causing severe cognitive and motor damage. Gene therapy for hemophilia also remains a viable option because of the amount of cell types that are capable of synthesizing biologically active FVIII and FIX following gene transfer using AAV vectors in vivo to correct hemophilia B (FIX deficiency), and it is considered an ideal target, as proven in preclinical studies. Recently, the clustered regularly interspaced palindromic repeats (CRISPR)/CRISPR-associated protein 9 gene-editing tool has taken a center stage in gene therapy research and is reported to be efficient and highly precise. The application of gene therapy to these areas has pushed forward the therapeutic clinical application.Odiba Arome SolomonOkoro Nkwachukwu OziamaraDurojaye Olanrewaju AyodejiWu YanjunDe Gruyterarticleclinical trialsgene therapyhemoglobinhemophilia bneurodegenerativeocularBiology (General)QH301-705.5ENOpen Life Sciences, Vol 16, Iss 1, Pp 431-441 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	clinical trials gene therapy hemoglobin hemophilia b neurodegenerative ocular Biology (General) QH301-705.5
spellingShingle	clinical trials gene therapy hemoglobin hemophilia b neurodegenerative ocular Biology (General) QH301-705.5 Odiba Arome Solomon Okoro Nkwachukwu Oziamara Durojaye Olanrewaju Ayodeji Wu Yanjun Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders
description	A new approach is adopted to treat primary immunodeficiency disorders, such as the severe combined immunodeficiency (SCID; e.g., adenosine deaminase SCID [ADA-SCID] and IL-2 receptor X-linked severe combined immunodeficiency [SCID-X1]). The success, along with the feasibility of gene therapy, is undeniable when considering the benefits recorded for patients with different classes of diseases or disorders needing treatment, including SCID-X1 and ADA-SCID, within the last two decades. β-Thalassemia and sickle cell anemia are two prominent monogenic blood hemoglobin disorders for which a solution has been sought using gene therapy. For instance, transduced autologous CD34+ HSCs via a self-inactivating (SIN)-Lentivirus (LV) coding for a functional copy of the β-globin gene has become a feasible procedure. adeno-associated virus (AAV) vectors have found application in ocular gene transfer in retinal disease gene therapy (e.g., Leber’s congenital amaurosis type 2), where no prior treatment existed. In neurodegenerative disorders, successes are now reported for cases involving metachromatic leukodystrophy causing severe cognitive and motor damage. Gene therapy for hemophilia also remains a viable option because of the amount of cell types that are capable of synthesizing biologically active FVIII and FIX following gene transfer using AAV vectors in vivo to correct hemophilia B (FIX deficiency), and it is considered an ideal target, as proven in preclinical studies. Recently, the clustered regularly interspaced palindromic repeats (CRISPR)/CRISPR-associated protein 9 gene-editing tool has taken a center stage in gene therapy research and is reported to be efficient and highly precise. The application of gene therapy to these areas has pushed forward the therapeutic clinical application.
format	article
author	Odiba Arome Solomon Okoro Nkwachukwu Oziamara Durojaye Olanrewaju Ayodeji Wu Yanjun
author_facet	Odiba Arome Solomon Okoro Nkwachukwu Oziamara Durojaye Olanrewaju Ayodeji Wu Yanjun
author_sort	Odiba Arome Solomon
title	Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders
title_short	Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders
title_full	Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders
title_fullStr	Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders
title_full_unstemmed	Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders
title_sort	gene therapy in pids, hemoglobin, ocular, neurodegenerative, and hemophilia b disorders
publisher	De Gruyter
publishDate	2021
url	https://doaj.org/article/375714475dc643baa2b39f505edeb293
work_keys_str_mv	AT odibaaromesolomon genetherapyinpidshemoglobinocularneurodegenerativeandhemophiliabdisorders AT okoronkwachukwuoziamara genetherapyinpidshemoglobinocularneurodegenerativeandhemophiliabdisorders AT durojayeolanrewajuayodeji genetherapyinpidshemoglobinocularneurodegenerativeandhemophiliabdisorders AT wuyanjun genetherapyinpidshemoglobinocularneurodegenerativeandhemophiliabdisorders
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Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders

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