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Gene Therapy in Tissue Engineering: Prospects and Challenges
Published in Rajesh K. Kesharwani, Raj K. Keservani, Anil K. Sharma, Tissue Engineering, 2022
It has been a long battle to ensure that stimulation of stem cells in situ should lead to specific regeneration and not to scar formation or teratogenesis. There are persistent challenges namely inability to sustain growth factors’ presence in the culture media for indefinite period of time, correct dosage, and lack of activity of recombinant factors. Thus, research nowadays is focused on overcoming the hurdles and transduce cell in vivo to bring about suitable regeneration processes by investigating “gene-activated matrices” using plasmids coding as delivery vehicles (Bonadio, 2000). Stems cells herald the backbone of tissue engineering, but they are also the critical targets for correcting any genetic defects. Many of the hazards caused by direct gene transfer in human are now been avoided by genetically modifying these cells ex vivo using viral and nonviral transducing agents (Asahara et al., 2000). Gene therapy is the answer to the successful cure of junctional epidermolysis bullosa, a debilitating skin disease by transducing epidermal cells (Dellambra et al., 2000). Undoubtedly, care should be taken to avoid mutagenesis by silencing the gene. Strategies such as site-directed mutagenesis and homologous recombination should be the crucial goal, though many intermediate methods can also be employed such as antisense RNA, RNA interference, and hammerhead and hairpin RNase(s) to preclude expression of mutant gene. Thus, in an attempt to revolutionize tissue engineering, frontiers of genetic engineering are being merged, so that reconstruction is now termed as “correction”. tissue engineering has a new definition as engineering of tissue function via stem cell-mediated gene therapy and holds tremendous prospects in clinical application. The next section of the chapter provides an understanding to the readers about gene therapy.
Investigation on skin-protective clothing that addresses needs of epidermolysis bullosa patients/children with epidermolysis bullosa and their parents
Published in The Journal of The Textile Institute, 2022
Ngan Yi Kitty Lam, Xue Luo, Li Li
The recruitment notice of participants was sent out to the patient database of the Taiwan Epidermolysis Bullosa Association. There were only six participants responded and willing to participate in this study. It is worth noting that there are difficulties to find patients who are willing to contribute to research because of their skin condition which needs professional medical care from time to time. Some of the skin medical condition is not suitable to travel outside and there would cause difficulties for public communication of both the patients and their parents. The six participants are all Mandarin native speakers and willing and motivated to participate in this study with informed consent. The definitive sample consisted of 2 children with EB who are 9 and 11 years old (Participants 1 and 2) at the time of the study, 1 female adult with EB who is 33 years old (Participant 3) at the time of the study and 3 parents of EB patients who range from 33 to 50 years old. The parents are a father of a 5 month-old male baby with EB (Participant 4), the mother of the 11 year-old female participant with EB (Participant 5); and the mother of a 17 year-old male youth with EB (Participant 6). Participant 1 and the 5 month-old male infant of Participant 4 were not diagnosed with any particular type of EB, while Participant 2 was diagnosed with Köebner subtype of EB simplex (EBS-K), Participant 3 with epidermolysis bullosa simplex (EBS), and the 17 year-old male child of Participant 6 with junctional epidermolysis bullosa.