Gene Therapy for Alopecia in Type II Rickets Model Rats Using Vitamin D Receptor-Expressing Adenovirus Vector

April 2023 in “ Research Square (Research Square) ”

Satoko Kise, Ayano Iijima, Chika Nagao, Tadashi Okada, Miyu Nishikawa, Shinichi Ikushiro, Teruyuki Nakanishi, Shigeto Sato, Kaori Yasuda, Toshiyuki Sakaki

gene therapy alopecia type II rickets vitamin D receptor adenovirus vector VDR gene Vdr-KO adenoviral vector hair growth cyst formation hair loss AdV VDR

Studysummary In this study, gene therapy using a VDR-expressing adenoviral vector significantly improved hair growth and reduced cyst formation in Vdr -KO rats with alopecia linked to type II rickets, whereas control treatments did not show these effects.

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The study investigated the use of gene therapy to treat alopecia in rats with type II rickets, a hereditary disease caused by a mutation in the vitamin D receptor (VDR) gene. Six 7-week-old female rats with a VDR gene knockout (Vdr-KO) were treated with an adenoviral vector (AdV) expressing the VDR gene. The treated rats showed significant VDR expression in the skin, enhanced hair growth, and low cyst formation, unlike the control groups. This suggests that gene therapy using a VDR-expressing AdV could be a potential treatment for alopecia associated with type II rickets.

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research Functional Analysis of Vitamin D Receptor Using Adenovirus Vector

4 citations , February 2023 in “Journal of steroid biochemistry and molecular biology/The Journal of steroid biochemistry and molecular biology”

In this study, only Vdr-knockout rats showed both abnormal skin formation and alopecia, which may provide insights into vitamin D receptor function and its role in the hair growth cycle.

research Functional Analyses of a Novel Missense and Other Mutations of the Vitamin D Receptor in Association with Alopecia

20 citations , July 2017 in “Scientific Reports”

This study found that a novel missense mutation in the vitamin D receptor caused hereditary 1,25-dihydroxyvitamin D-resistant rickets with alopecia by disrupting protein function, highlighting the importance of DNA binding in hair development.

research Mutations in the Vitamin D Receptor and Hereditary Vitamin D-Resistant Rickets

107 citations , March 2014 in “BoneKEy Reports”

This abstract reviews hereditary vitamin D-resistant rickets, a rare genetic condition causing severe early childhood rickets, and reports no new results; effective treatment typically requires high doses of calcium to address hypocalcemia and secondary hyperparathyroidism.

research The Role of Vitamin D Receptor Mutations in the Development of Alopecia

109 citations , June 2011 in “Molecular and Cellular Endocrinology”

Vitamin D receptor mutations can cause alopecia by affecting hair growth genes.

research Lymphoid Enhancer-Binding Factor-1 Interacts with the DNA-Binding Domain of the Vitamin D Receptor

37 citations , April 2011 in “Journal of Biological Chemistry”

This study discovered a novel interaction between the vitamin D receptor and LEF1, essential for normal Wnt signaling in keratinocytes, which is crucial for regular hair cycling.

research Disruption of the Hedgehog Signaling Pathway Contributes to Hair Follicle Cycling Deficiency in VDR Knockout Mice

53 citations , May 2010 in “Journal of Cellular Physiology”

This study found that mice lacking the Vitamin D receptor showed disrupted hair follicle cycling, which was partially restored with hedgehog signaling pathway activation, suggesting a role for this pathway in follicle regulation independent of vitamin D.

research Vitamin D and Human Health: Lessons from Vitamin D Receptor Null Mice

1533 citations , October 2008 in “Endocrine reviews”

This research highlights that engineered mice lacking the vitamin D receptor show multiple health issues similar to human vitamin D deficiency, underscoring the widespread impacts of the vitamin D endocrine system.

research Physical And Functional Interaction Between The Vitamin D Receptor And Hairless Corepressor, Two Proteins Required For Hair Cycling

215 citations , September 2003 in “Journal of Biological Chemistry”

This study found that the hairless gene product (Hr) suppresses VDR-mediated gene activation by directly interacting with the vitamin D receptor, potentially affecting hair follicle function.