J
Janet Lee
Researcher at National Institutes of Health
Publications - 32
Citations - 1592
Janet Lee is an academic researcher from National Institutes of Health. The author has contributed to research in topics: Haematopoiesis & Genetic enhancement. The author has an hindex of 18, co-authored 32 publications receiving 1318 citations. Previous affiliations of Janet Lee include University of California, Los Angeles & Jules Stein Eye Institute.
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Journal ArticleDOI
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
Suk See De Ravin,Xiaolin Wu,Susan Moir,Lela Kardava,Sandra Anaya-O'Brien,Nana Kwatemaa,Patricia Littel,Narda Theobald,Uimook Choi,Ling Su,Martha Marquesen,Dianne Hilligoss,Janet Lee,Clarissa M. Buckner,Kol A. Zarember,Geraldine M. O’Connor,Daniel W. McVicar,Douglas B. Kuhns,Robert E. Throm,Sheng Zhou,Luigi D. Notarangelo,I. Celine Hanson,M.J. Cowan,Elizabeth M. Kang,Coleen Hadigan,Michael M Meagher,John T. Gray,Brian P. Sorrentino,Harry L. Malech +28 more
TL;DR: It is reported that lentiviral gene therapy with nonmyeloablative conditioning corrected multiple immune cell types in five older SCID-X1 patients whose symptoms persisted despite undergoing hematopoietic stem cell transplant(s) as infants.
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CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease.
Suk See De Ravin,Linhong Li,Xiaolin Wu,Uimook Choi,Cornell Allen,Sherry Koontz,Janet Lee,Narda Theobald-Whiting,Jessica Chu,Mary Garofalo,Colin L. Sweeney,Lela Kardava,Susan Moir,Angelia Viley,Pachai Natarajan,Ling Su,Douglas B. Kuhns,Kol A. Zarember,Madhusudan Peshwa,Harry L. Malech +19 more
TL;DR: It is reported that CRISPR, a DNA editing technology, corrected blood stem cells from patients with an immunodeficiency disorder (chronic granulomatous disease) caused by mutations in NOX2, and gene repair of CD34+ hematopoietic stem and progenitor cells (HSPCs) may avoid problems associated with gene therapy.
Journal ArticleDOI
Arabidopsis Cryptochrome 2 Completes Its Posttranslational Life Cycle in the Nucleus
Xuhong Yu,John Klejnot,Xiaoying Zhao,Dror Shalitin,Maskit Maymon,Hongyun Yang,Janet Lee,Xuanming Liu,Javier Lopez,Chentao Lin +9 more
TL;DR: The results show that CRY2 mediates blue light inhibition of hypocotyl elongation and photoperiodic promotion of floral initiation in the nucleus, and it is demonstrated that a photoreceptor can complete its posttranslational life cycle inside the nucleus.
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Targeted gene addition in human CD34 + hematopoietic cells for correction of X-linked chronic granulomatous disease
Suk See De Ravin,Andreas Reik,Pei-Qi Liu,Linhong Li,Xiaolin Wu,Ling Su,Castle Raley,Narda Theobald,Uimook Choi,Alexander H. Song,Andy Chan,Jocelynn R. Pearl,David Paschon,Janet Lee,Hannah Newcombe,Sherry Koontz,Colin L. Sweeney,David A. Shivak,Kol A. Zarember,Madhusudan Peshwa,Philip D. Gregory,Fyodor D. Urnov,Harry L. Malech +22 more
TL;DR: It is demonstrated that temporally optimized delivery of zinc finger nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constructs in human HSPCs approaches clinically relevant levels of TI into the AAVS1 safe harbor locus.
Journal ArticleDOI
Complement factor H genotypes impact risk of age-related macular degeneration by interaction with oxidized phospholipids
Peter X. Shaw,Li Zhang,Ming Zhang,Hongjun Du,Hongjun Du,Ling Zhao,Ling Zhao,Clara Lee,Seanna Grob,Siok Lam Lim,Guy Hughes,Janet Lee,Matthew Bedell,Mark H. Nelson,Fang Lu,Martin Krupa,Jing Luo,Hong Ouyang,Zhidan Tu,Zhiguang Su,Jin Zhu,Jin Zhu,Xinran Wei,Xinran Wei,Zishan Feng,Yaou Duan,Yaou Duan,Zhenglin Yang,Henry Ferreyra,Dirk-Uwe Bartsch,Igor Kozak,Liangfang Zhang,Feng Lin,Hui Sun,Hong Feng,Kang Zhang,Kang Zhang +36 more
TL;DR: It is demonstrated that CFH binds to oxPLs and that oxPL stimulation increases expression of genes involved in macrophage infiltration, inflammation, and neovascularization in the eye.