scholarly journals Production of transgenic swine by sperm stem cell transplantation and gene editing technology

2019 ◽  
Author(s):  
Tianzi Zhang
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Gene Editing ◽  
Transgenic Swine

Inherited immunohematological and metabolic diseases have the potential to improve significantly, or be cured, using haematopoietic stem cell transplantation gene therapy

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Gene Editing ◽  
Metabolic Diseases ◽  
Haematopoietic Stem Cell ◽  
Specific Gene ◽  
Neurometabolic Disorders

inherited immunohematological and metabolic diseases have the potential to improve significantly, or be cured, using haematopoietic stem cell transplantation (HSPC) gene therapy Until autologous HSPC gene therapy makes it to the clinic, a lot of financial and logistical issues must be resolved. Conventional methods make use of centralized production facilities and a small number of specialist treatment facilities for sickness and gene therapy, but totally automated transduction may make this paradigm obsolete. The use of alkylating medications for conditioning is connected to both short-and long-term negative outcomes. The adoption of antibody-based conditioning regimens that target molecules expressed on host haematopoietic cells, such as CD117 or CD45, might allow for a decrease in alkylating agent dosage.Preclinical research has suggested that direct intravascular distribution of viral vectors would be a good alternative to ex vivo gene transfer, however with limited efficacy. A therapeutic impact and unwanted transgenic-related damage is occasionally sought. Locus-specific gene editing may be able to make use of naturally occurring gene control mechanisms. On the other hand, synthetic minigenes may not always reproduce gene expression in cells and may require complicated design. The improved efficiency in gene editing will almost probably lead to an increase in the number of therapeutic applications for gene-edited HSPCs. Although gene editing is theoretically safer than vector-based gene addition approaches, the clinical safety of HSPC gene therapy has yet to be proved. Double-strand breaks can create rearrangements such as deletions, inversions, and translocations, but Cas9 nucleases might suppress any residual issues. Base editing and prime editing can increase genome engineering safety and efficiency. In gene therapy, much further development is necessary before gene editing may be applied in a clinical-scale setting. Clinical translation of complex cellular therapies is aided by previous advances in vector-based gene therapy, which has opened the way for the area of genome editing. A gene editing evaluation needs alterations in tests and methodologies.Most HSPC gene therapies have been utilized to address specific genetic diseases. The longevity and safety of HSPC gene therapy may increase, thereby presenting the possibility of addressing new illness conditions. Consider the scenario of treatment that may last for a long time if HSPC-derived microglia was utilized. Biological systemic treatment delivery and/or elimination of blood disease reservoirs might potentially enhance chronic infectious diseases and cancer. The chimeric antigen receptors of T cells, for example, can cure several forms of haematological cancers. HSPC gene therapy will increase greatly in the future, addressing a larger spectrum of immune haematological and neurometabolic disorders.

Caregiver perspectives on psychosocial care in pediatric hematopoietic stem cell transplantation (HCT).

2020 ◽  
Vol 8 (1) ◽  
pp. 67-78 ◽  
Author(s):  
Anne E. Kazak ◽  
Avi Madan Swain ◽  
Ahna L. H. Pai ◽  
Kimberly Canter ◽  
Olivia Carlson ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Psychosocial Care ◽  
Hematopoietic Stem
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