VanLith相关文献与解析，生知库 | 链接生命科学的每一步

Liu, Yaming; Hassan, Hind; Brooks, Tayla R; VanLith, Caitlin; Cooley, Matthew; Elgozair, Mohamad; Ahmed, Fowsiyo Y; Giama, Nasra H; Campbell, Nellie A; Su, Lianyong; Tai, Yun-Ling; Ren, Jianlin; Zhou, Huiping; Roberts, Lewis R

肿瘤

胆管癌

肿瘤免疫

代谢

发表日期：2025

文献求助收藏

In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

利用体内慢病毒载体基因疗法治愈1型遗传性酪氨酸血症并预防癌前病变和癌变的发展

期刊:Nature Communications

影响因子:15.7

doi:10.1038/s41467-022-32576-7

Nicolas, Clara T; VanLith, Caitlin J; Hickey, Raymond D; Du, Zeji; Hillin, Lori G; Guthman, Rebekah M; Cao, William J; Haugo, Benjamin; Lillegard, Annika; Roy, Diya; Bhagwate, Aditya; O'Brien, Daniel; Kocher, Jean-Pierre; Kaiser, Robert A; Russell, Stephen J; Lillegard, Joseph B

Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

使用腺相关病毒血清型 Anc80 在小鼠模型中提供苯丙酮尿症的持久治愈

期刊:Journal of Inherited Metabolic Disease

影响因子:4.2

doi:10.1002/jimd.12392

Robert A Kaiser, Nicholas D Weber, Laia Trigueros-Motos, Kari L Allen, Michael Martinez, William Cao, Caitlin J VanLith, Lori G Hillin, Anne Douar, Gloria González-Aseguinolaza, Rafael Aldabe, Joseph B Lillegard

The Combination of Venetoclax and Ixazomib Selectively and Efficiently Kills HIV-Infected Cell Lines but Has Unacceptable Toxicity in Primary Cell Models

维奈克拉与伊沙佐米的组合能够选择性且有效地杀死 HIV 感染细胞系，但在原代细胞模型中却具有不可接受的毒性

期刊:Journal of Virology

影响因子:4

doi:10.1128/JVI.00138-21

Alecia Alto #, Sekar Natesampillai #, Aswath P Chandrasekar, Ashton Krogman, Anisha Misra, Fnu Shweta, Caitlin VanLith, Joseph D Yao, Nathan W Cummins, Andrew D Badley

Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing

开发具有人源化 R408W 突变的苯丙酮尿症猪模型以进行基因编辑

期刊:PLoS One

影响因子:2.9

doi:10.1371/journal.pone.0245831

Robert A Kaiser, Daniel F Carlson, Kari L Allen, Dennis A Webster, Caitlin J VanLith, Clara T Nicolas, Lori G Hillin, Yue Yu, Catherine W Kaiser, William R Wahoff, Raymond D Hickey, Adrienne L Watson, Shelley R Winn, Beat Thöny, Douglas R Kern, Cary O Harding, Joseph B Lillegard

The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism

基因靶向治疗在遗传性酪氨酸血症1型（一种先天性代谢缺陷的主要适应症）中的应用前景

期刊:Expert Opinion on Orphan Drugs

影响因子:0.8

doi:10.1080/21678707.2020.1791082

Thompson, Whitney S; Mondal, Gourish; Vanlith, Caitlin J; Kaiser, Robert A; Lillegard, Joseph B

代谢

发表日期：2020

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Ex Vivo Cell Therapy by Ectopic Hepatocyte Transplantation Treats the Porcine Tyrosinemia Model of Acute Liver Failure

异位肝细胞移植体外细胞疗法治疗猪酪氨酸血症急性肝衰竭模型

期刊:Molecular Therapy-Methods & Clinical Development

影响因子:

doi:10.1016/j.omtm.2020.07.009

Clara T Nicolas, Robert A Kaiser, Raymond D Hickey, Kari L Allen, Zeji Du, Caitlin J VanLith, Rebekah M Guthman, Bruce Amiot, Lukkana Suksanpaisan, Bing Han, Maria Giovanna Francipane, Amin Cheikhi, Huailei Jiang, Aditya Bansal, Mukesh K Pandey, Ishan Garg, Val Lowe, Aditya Bhagwate, Daniel O'Brien,

Ex Vivo Hepatocyte Reprograming Promotes Homology-Directed DNA Repair to Correct Metabolic Disease in Mice After Transplantation

体外肝细胞重编程促进同源定向DNA修复，从而纠正移植后小鼠的代谢性疾病

期刊:Hepatology Communications

影响因子:5.6

doi:10.1002/hep4.1315

Caitlin J VanLith ，Rebekah M Guthman ，Clara T Nicolas ，Kari L Allen ，Yuanhang Liu ，Jennifer A Chilton ，Zachariah P Tritz ，Scott L Nyberg ，Robert A Kaiser ，Joseph B Lillegard ，Raymond D Hickey

Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1

自体基因和细胞疗法在大型猪遗传性酪氨酸血症1型模型中提供安全且长期的治愈性治疗

期刊:Cell Transplantation

影响因子:3.2

doi:10.1177/0963689718814188

Hickey, Raymond D; Nicolas, Clara T; Allen, Kari; Mao, Shennen; Elgilani, Faysal; Glorioso, Jaime; Amiot, Bruce; VanLith, Caitlin; Guthman, Rebekah; Du, Zeji; Chen, Harvey; Harding, Cary O; Kaiser, Robert A; Nyberg, Scott L; Lillegard, Joseph B

细胞生物学

发表日期：2019

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Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1

在1型遗传性酪氨酸血症小鼠模型中，体外肝细胞定向基因编辑的治愈性治疗

期刊:Human Gene Therapy

影响因子:4

doi:10.1089/hum.2017.252

VanLith, Caitlin; Guthman, Rebekah; Nicolas, Clara T; Allen, Kari; Du, Zeji; Joo, Dong Jin; Nyberg, Scott L; Lillegard, Joseph B; Hickey, Raymond D

Blood and tissue dysregulated bile acids and short-chain fatty acids in cholangiocarcinoma

胆管癌中血液和组织中胆汁酸和短链脂肪酸的失调

In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

利用体内慢病毒载体基因疗法治愈1型遗传性酪氨酸血症并预防癌前病变和癌变的发展

Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

使用腺相关病毒血清型 Anc80 在小鼠模型中提供苯丙酮尿症的持久治愈

The Combination of Venetoclax and Ixazomib Selectively and Efficiently Kills HIV-Infected Cell Lines but Has Unacceptable Toxicity in Primary Cell Models

维奈克拉与伊沙佐米的组合能够选择性且有效地杀死 HIV 感染细胞系，但在原代细胞模型中却具有不可接受的毒性

Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing

开发具有人源化 R408W 突变的苯丙酮尿症猪模型以进行基因编辑

The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism

基因靶向治疗在遗传性酪氨酸血症1型（一种先天性代谢缺陷的主要适应症）中的应用前景

Ex Vivo Cell Therapy by Ectopic Hepatocyte Transplantation Treats the Porcine Tyrosinemia Model of Acute Liver Failure

异位肝细胞移植体外细胞疗法治疗猪酪氨酸血症急性肝衰竭模型

Ex Vivo Hepatocyte Reprograming Promotes Homology-Directed DNA Repair to Correct Metabolic Disease in Mice After Transplantation

体外肝细胞重编程促进同源定向DNA修复，从而纠正移植后小鼠的代谢性疾病

Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1

自体基因和细胞疗法在大型猪遗传性酪氨酸血症1型模型中提供安全且长期的治愈性治疗

Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1

在1型遗传性酪氨酸血症小鼠模型中，体外肝细胞定向基因编辑的治愈性治疗