Research Topic

HSCT (Hematopoietic stem cell transplantation) and Gene Therapy in Non-Malignant Disorders

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About this Research Topic

General pediatricians and pediatric hematologists frequently face patients with rare non-malignant conditions. Albeit in the last decades allogeneic hematopoietic stem cell transplantation (HSCT) represented the only available treatment able to cure many of these disorders (with variable success rates), advances in vector design, ex-vivo cell manipulation as well as improvement in preparative regimens and patient care led to the development of the first clinical trials of ex-vivo gene therapy. Today, several of these monogenic non-malignant diseases have different treatment options resulting in an increasing and ongoing debate on which is the best therapeutic option.

The goal of this Research Topic is to give a comprehensive overview (including strengths and weaknesses) of two different treatment options (allogeneic HSCT and ex-vivo gene therapy) for the diseases for which they are available (or almost ready to be brought into the clinic). In particular, to stimulate discussion and to make this similar to a public debate, two companion papers touching the same points (i.e., milestones of the procedure, major results, actual indications, pros and cons, 5-year perspective) from different experts in the field will be presented for each disease.

We welcome the following themes:
• ADA-SCID and X-SCID
• Wiskott-Aldrich syndrome
• Chronic Granulomatous Disease
• Thalassemia
• Sickle cell disease
• X-linked adrenoleukodistrophy
• Metachromatic leukodystrophy
• Mucopolysaccharidosis
• Fanconi anemia

Moreover, spontaneous original manuscript commentary or case reports will be considered.

Topic Editor Dr. Robert Chiesa has been part of the advisory boards for Orchard, Bluebirdbio and Gilead. All other Topic Editors declare no competing interests with regards to the Research Topic subject.


Keywords: HSCT, Gene therapy


Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.

General pediatricians and pediatric hematologists frequently face patients with rare non-malignant conditions. Albeit in the last decades allogeneic hematopoietic stem cell transplantation (HSCT) represented the only available treatment able to cure many of these disorders (with variable success rates), advances in vector design, ex-vivo cell manipulation as well as improvement in preparative regimens and patient care led to the development of the first clinical trials of ex-vivo gene therapy. Today, several of these monogenic non-malignant diseases have different treatment options resulting in an increasing and ongoing debate on which is the best therapeutic option.

The goal of this Research Topic is to give a comprehensive overview (including strengths and weaknesses) of two different treatment options (allogeneic HSCT and ex-vivo gene therapy) for the diseases for which they are available (or almost ready to be brought into the clinic). In particular, to stimulate discussion and to make this similar to a public debate, two companion papers touching the same points (i.e., milestones of the procedure, major results, actual indications, pros and cons, 5-year perspective) from different experts in the field will be presented for each disease.

We welcome the following themes:
• ADA-SCID and X-SCID
• Wiskott-Aldrich syndrome
• Chronic Granulomatous Disease
• Thalassemia
• Sickle cell disease
• X-linked adrenoleukodistrophy
• Metachromatic leukodystrophy
• Mucopolysaccharidosis
• Fanconi anemia

Moreover, spontaneous original manuscript commentary or case reports will be considered.

Topic Editor Dr. Robert Chiesa has been part of the advisory boards for Orchard, Bluebirdbio and Gilead. All other Topic Editors declare no competing interests with regards to the Research Topic subject.


Keywords: HSCT, Gene therapy


Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.

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