Aikawa E et al (2015) Systemic high-mobility group box 1 administration suppresses skin inflammation by inducing an accumulation of PDGFRα+ mesenchymal cells from bone marrow. Sci Rep 5(1):1–13

Article  Google Scholar 

Alexeev V, Uitto J, Igoucheva O (2011) Gene expression signatures of mouse bone marrow-derived mesenchymal stem cells in the cutaneous environment and therapeutic implications for blistering skin disorder. Cytotherapy 13(1):30–45

Article  CAS  PubMed  Google Scholar 

Alvin G (2021) Cell therapy: the new approach to der-matology and dermatologic surgery. Clin Surg 5(8):1–14

Google Scholar 

Angelova-Fischer I et al (2005) Kindler syndrome: a case report and proposal for clinical diagnostic criteria. Acta Dermatovenerol Alp Panonica Adriat 14:61–67

Google Scholar 

Arabi F, Mansouri V, Ahmadbeigi N (2022) Gene therapy clinical trials, where do we go? An overview. Biomed Pharmacother 153:113324

Article  CAS  PubMed  Google Scholar 

Arin MJ (2009) The molecular basis of human keratin disorders. Hum Genet 125(4):355–373

Article  MathSciNet  CAS  PubMed  Google Scholar 

Ashton G (2004) Kindler syndrome. Clin Exp Dermatol 29(2):116–121

Article  CAS  PubMed  Google Scholar 

Ashton G et al (2001) α6β4 integrin abnormalities in junctional epidermolysis bullosa with pyloric atresia. Br J Dermatol 144(2):408–414

Article  CAS  PubMed  Google Scholar 

Atkinson SD et al (2011) Development of allele-specific therapeutic siRNA for keratin 5 mutations in epidermolysis bullosa simplex. J Investig Dermatol 131(10):2079–2086

Article  CAS  PubMed  Google Scholar 

Aumailley M et al (2005) A simplified laminin nomenclature. Matrix Biol 24(5):326–332

Article  CAS  PubMed  Google Scholar 

Aushev M et al (2017) Traceless targeting and isolation of gene-edited immortalized keratinocytes from epidermolysis bullosa simplex patients. Mol Ther Methods Clin Dev 6:112–123

Article  CAS  PubMed  PubMed Central  Google Scholar 

Baardman R et al (2021) Novel insights into the epidemiology of epidermolysis bullosa (EB) from the Dutch EB Registry: EB more common than previously assumed? J Eur Acad Dermatol Venereol 35(4):995–1006

Article  CAS  PubMed  Google Scholar 

Balistreri CR et al (2020) Stem cell therapy: old challenges and new solutions. Mol Biol Rep 47(4):3117–3131

Article  CAS  PubMed  Google Scholar 

Bardhan A et al (2020) Epidermolysis bullosa. Nat Rev Dis Primers 6(1):1–27

Article  Google Scholar 

Bartel RL (2015) Stem cells and cell therapy: autologous cell manufacturing. In: Translational regenerative medicine. Elsevier, pp 107–112

Chapter  Google Scholar 

Bartoszewski R, Sikorski AF (2019) Editorial focus: understanding off-target effects as the key to successful RNAi therapy. Cell Mol Biol Lett 24(1):1–23

Article  Google Scholar 

Bauer J et al (2017) Closure of a large chronic wound through transplantation of gene-corrected epidermal stem cells. J Invest Dermatol 137(3):778–781

Article  CAS  PubMed  Google Scholar 

Baum C et al (2003) Side effects of retroviral gene transfer into hematopoietic stem cells. Blood, J Am Soc Hematol 101(6):2099–2113

CAS  Google Scholar 

Benati D et al (2018) CRISPR/Cas9-mediated in situ correction of LAMB3 gene in keratinocytes derived from a junctional epidermolysis bullosa patient. Mol Ther 26(11):2592–2603

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bennett CF, Swayze EE (2010) RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol 50:259–293

Article  CAS  PubMed  Google Scholar 

Bolling M et al (2011) Mutations in KRT5 and KRT14 cause epidermolysis bullosa simplex in 75% of the patients. Br J Dermatol 164(3):637–644

CAS  PubMed  Google Scholar 

Bonaventura G et al (2021) Stem cells: innovative therapeutic options for neurodegenerative diseases? Cells 10(8):1992

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bordet T, Behar-Cohen F (2019) Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives. Drug Discov Today 24(8):1685–1693

Article  CAS  PubMed  Google Scholar 

Bordignon C et al (1999) Cell therapy: achievements and perspectives. Haematologica 84(12):1110–1149

CAS  PubMed  Google Scholar 

Bornert O et al (2016) Analysis of the functional consequences of targeted exon deletion in COL7A1 reveals prospects for dystrophic epidermolysis bullosa therapy. Mol Ther 24(7):1302–1311

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bornert O et al (2021) QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa: a preclinical study. J Investig Dermatol 141(4):883–893

Article  CAS  PubMed  Google Scholar 

Bremer J et al (2016) Antisense oligonucleotide-mediated exon skipping as a systemic therapeutic approach for recessive dystrophic epidermolysis bullosa. Mol Ther Nucleic Acids:5

Bruckner AL et al (2020) The challenges of living with and managing epidermolysis bullosa: insights from patients and caregivers. Orphanet J Rare Dis 15(1):1–14

Article  PubMed  PubMed Central  Google Scholar 

Bruckner-Tuderman L et al (2013) Progress in epidermolysis bullosa research: summary of DEBRA International Research Conference 2012. J Investig Dermatol 133(9):2121–2126

Article  CAS  PubMed  Google Scholar 

Carter DM et al (1987) Treatment of junctional epidermolysis bullosa with epidermal autografts. J Am Acad Dermatol 17(2):246–250

Article  CAS  PubMed  Google Scholar 

Chino T et al (2008) Bone marrow cell transfer into fetal circulation can ameliorate genetic skin diseases by providing fibroblasts to the skin and inducing immune tolerance. Am J Pathol 173(3):803–814

Article  CAS  PubMed  PubMed Central  Google Scholar 

Christiano AM et al (1994) Cloning of human type VII collagen. Complete primary sequence of the alpha 1 (VII) chain and identification of intragenic polymorphisms. J Biol Chem 269(32):20256–20262

Article  CAS  PubMed  Google Scholar 

Cogan J et al (2014) Aminoglycosides restore full-length type VII collagen by overcoming premature termination codons: therapeutic implications for dystrophic epidermolysis bullosa. Mol Ther 22(10):1741–1752

Article  CAS  PubMed  PubMed Central  Google Scholar 

Coluccio A et al (2013) Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. Mol Ther 21(9):1695–1704

Article  CAS  PubMed  PubMed Central  Google Scholar 

Condrat I et al (2019) Junctional epidermolysis bullosa: allelic heterogeneity and mutation stratification for precision medicine. Front Med 5:363

Article  Google Scholar 

Cong L et al (2013) Multiplex genome engineering using CRISPR/Cas systems. Science 339(6121):819–823

Article  CAS  PubMed  PubMed Central  Google Scholar 

Conget P et al (2010) Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa. Cytotherapy 12(3):429–431

Article  CAS  PubMed  Google Scholar 

Coulombe PA, Kerns ML, Fuchs E (2009) Epidermolysis bullosa simplex: a paradigm for disorders of tissue fragility. J Clin Invest 119(7):1784–1793

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cutlar L, Greiser U, Wang W (2014) Gene therapy: pursuing restoration of dermal adhesion in recessive dystrophic epidermolysis bullosa. Exp Dermatol 23(1):1–6

Article  CAS  PubMed  Google Scholar 

Dang N, Murrell DF (2008) Mutation analysis and characterization of COL7A1 mutations in dystrophic epidermolysis bullosa. Exp Dermatol 17(7):553–568

Article  CAS  PubMed  Google Scholar 

Das BB, Sahoo S (2004) Dystrophic epidermolysis bullosa. J Perinatol 24(1):41–47

Article  PubMed