Thomas Sandmann

denali therapeutics

H指数:29

被引数:4,594

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Microglial TDP-43 mediates myelin refinement and represses Tyrobp cryptic exon inclusion in mice

小胶质细胞中的TDP-43介导髓鞘修剪并抑制小鼠中Tyrobp内含子的隐藏外显子包含

Nature Neuroscience

IF20

2026-07-080

OAAI

Author Correction: Fc-engineered large molecules targeting blood-brain barrier transferrin receptor and CD98hc have distinct central nervous system and peripheral biodistribution

作者更正：Fc工程化大分子靶向血脑屏障转铁蛋白受体和CD98hc具有不同的中枢神经系统和外周生物分布

Nature Communications

IF15.7

2026-04-150

OAAI

Nathalie Khoury; Michelle E. Pizzo; Claire B. Discenza; David Joy; David Tatarakis; Mihail Ivilinov Todorov; Moritz Negwer; Connie Ha; Gabrielly L. De Melo; Lily Sarrafha; Matthew J. Simon; Darren Chan; Roni Chau; Kylie S. Chew; Johann Chow; Allisa Clemens; Yaneth Robles-Colmenares; Jason C. Dugas; Joseph Duque; Doris Kaltenecker; Holly Kane; Amy Leung; Edwin Lozano; Arash Moshkforoush; Elysia Roche; Thomas Sandmann; Mabel Tong; Kaitlin Xa; Yinhan Zhou; Joseph W. Lewcock; Ali Ertürk; Robert G. Thorne; Meredith E. K. Calvert; Y. Joy Yu Zuchero

Investigational eIF2B activator DNL343 modulates the integrated stress response in preclinical models of TDP-43 pathology and individuals with ALS in a randomized clinical trial

实验性eIF2B激活剂DNL343在TDP-43病理学的临床前模型和随机临床试验中的ALS患者中调节整合应激反应

Nature Communications

IF15.7

2025-08-180

OAAI

Brittany N. Flores; Seungyoon B. Yu; Isaac V. Cohen; Melania H. Fanok; Wei Luan; Romeo D. Maciuca; Linus D. Sun; Richard M. Tsai; Maurits Vissers; Lars Smits; Tommy M. Bunte; Anna Bakardjiev; Srijana Balasundar; Meredith E. K. Calvert; Marcus Y. Chin; Sarah K. Dobbins; William E. Dowdle; Meng Fang; Jules A. A. C. Heuberger; Connie L. Ha; Fen Huang; Takashi Miyamoto; Maksim Osipov; Lidia Madrid San Martin; Katie Saund; David Tatarakis; Anthony Q. Vu; Chenling Xiong; Gene W. Yeo; Geert Jan Groeneveld; Leonard H. van den Berg; Shyeilla Dhuria; Anthony A. Estrada; Danna Jennings; Thomas Sandmann; Carole Ho; Kimberly Scearce-Levie; Ernie Yulyaningsih; Adam K. Walker; Gilbert Di Paolo; Lesley A. Kane; Matthew D. Troyer; Joseph W. Lewcock

Fc-engineered large molecules targeting blood-brain barrier transferrin receptor and CD98hc have distinct central nervous system and peripheral biodistribution

N. Khoury, M. E. Pizzo, C. B. Discenza, D. Joy, D. Tatarakis, M. I. Todorov, M. Negwer, C. Ha, G. L. De Melo, L. Sarrafha, M. J. Simon, D. Chan, R. Chau, K. S. Chew, J. Chow, A. Clemens, Y. Robles-Colmenares, J. C. Dugas, J. Duque, D. Kaltenecker, H. Kane, A. Leung, E. Lozano, A. Moshkforoush, E. Roche, T. Sandmann, M. Tong, K. Xa, Y. Zhou, J. W. Lewcock, A. Ertürk, R. G. Thorne, M. E. K. Calvert, Y. J. Yu Zuchero, Fc工程化大分子靶向血脑屏障转铁蛋白受体和CD98hc具有不同的中枢神经系统和外周生物分布。Nat. Commun. 16, 1822 (2025)。

Nature Communications

IF15.7

2025-02-200

OAAI

DNL343 is an investigational CNS penetrant eukaryotic initiation factor 2B activator that prevents and reverses the effects of neurodegeneration caused by the integrated stress response

ELIFE

IF0

2024-09-171

Yulyaningsih, Ernie; Suh, Jung H.; Fanok, Melania; Chau, Roni; Solanoy, Hilda; Takahashi, Ryan; Bakardjiev, Anna, I; Becerra, Isabel; Benitez, N. Butch; Chiu, Chi-Lu; Davis, Sonnet S.; Dowdle, William E.; Earr, Timothy; Estrada, Anthony A.; Gill, Audrey; Ha, Connie; Haddick, Patrick C. G.; Henne, Kirk R.; Larhammar, Martin; Leung, Amy W-S; Maciuca, Romeo; Memarzadeh, Bahram; Nguyen, Hoang N.; Nugent, Alicia A.; Osipov, Maksim; Ran, Yingqing; Rebadulla, Kevin; Roche, Elysia; Sandman, Thomas; Wang, Jing; Lewcock, Joseph W.; Scearce-Levie, Kimberly; Kane, Lesley A.; Sanchez, Pascal E.

Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier

SCIENCE TRANSLATIONAL MEDICINE

IF14.6

2024-08-145

PRE

Barker, Scarlett J.; Thayer, Mai B.; Kim, Chaeyoung; Tatarakis, David; Simon, Matthew J.; Dial, Rebekah; Nilewski, Lizanne; Wells, Robert C.; Zhou, Yinhan; Afetian, Megan; Akkapeddi, Padma; Chappell, Alfred; Chew, Kylie S.; Chow, Johann; Clemens, Allisa; Discenza, Claire B.; Dugas, Jason C.; Dwyer, Chrissa; Earr, Timothy; Ha, Connie; Ho, Yvonne S.; Huynh, David; Lozano, Edwin I.; Jayaraman, Srini; Kwan, Wanda; Mahon, Cathal; Pizzo, Michelle; Robles-Colmenares, Yaneth; Roche, Elysia; Sanders, Laura; Stergioulis, Alexander; Tong, Raymond; Tran, Hai; Zuchero, Joy Yu; Estrada, Anthony A.; Gadkar, Kapil; Koth, Christopher M. M.; Sanchez, Pascal E.; Thorne, Robert G.; Watts, Ryan J.; Sandmann, Thomas; Kane, Lesley A.; Rigo, Frank; Dennis, Mark S.; Lewcock, Joseph W.; Devos, Sarah L.

Peripheral expression of brain-penetrant progranulin rescues pathologies in mouse models of frontotemporal lobar degeneration

SCIENCE TRANSLATIONAL MEDICINE

IF14.6

2024-06-050

PRE

The Genetic Drivers of Juvenile, Young, and Early-Onset Parkinson's Disease in India

印度青少年，年轻和早发性帕金森氏病的遗传驱动因素

MOVEMENT DISORDERS

IF7.6

2023-11-2810

OAAI

Andrews, Shan V.; Kukkle, Prashanth L.; Menon, Ramesh; Geetha, Thenral S.; Goyal, Vinay; Kandadai, Rukmini Mridula; Kumar, Hrishikesh; Borgohain, Rupam; Mukherjee, Adreesh; Wadia, Pettarusp M.; Yadav, Ravi; Desai, Soaham; Kumar, Niraj; Joshi, Deepika; Murugan, Sakthivel; Biswas, Atanu; Pal, Pramod K.; Oliver, Merina; Nair, Sandhya; Kayalvizhi, Anbu; Samson, Praveena L.; Deshmukh, Manjari; Bassi, Akshi; Sandeep, Charugulla; Mandloi, Nitin; Davis, Oliver B.; Roberts, Melissa A.; Leto, Dara E.; Henry, Anastasia G.; Di Paolo, Gilbert; Muthane, Uday; Das, Shymal K.; Peterson, Andrew S.; Sandmann, Thomas; Gupta, Ravi; Ramprasad, Vedam L.

Novel App knock-in mouse model shows key features of amyloid pathology and reveals profound metabolic dysregulation of microglia

MOLECULAR NEURODEGENERATION

IF17.5

2022-06-1146

OAAI

Xia, Dan; Lianoglou, Steve; Sandmann, Thomas; Calvert, Meredith; Suh, Jung H.; Thomsen, Elliot; Dugas, Jason; Pizzo, Michelle E.; DeVos, Sarah L.; Earr, Timothy K.; Lin, Chia-Ching; Davis, Sonnet; Ha, Connie; Leung, Amy Wing-Sze; Hoang Nguyen; Chau, Roni; Yulyaningsih, Ernie; Lopez, Isabel; Solanoy, Hilda; Masoud, Shababa T.; Liang, Chun-chi; Lin, Karin; Astarita, Giuseppe; Khoury, Nathalie; Zuchero, Joy Yu; Thorne, Robert G.; Shen, Kevin; Miller, Stephanie; Palop, Jorge J.; Garceau, Dylan; Sasner, Michael; Whitesell, Jennifer D.; Harris, Julie A.; Hummel, Selina; Gnorich, Johannes; Wind, Karin; Kunze, Lea; Zatcepin, Artem; Brendel, Matthias; Willem, Michael; Haass, Christian; Barnett, Daniel; Zimmer, Till S.; Orr, Anna G.; Scearce-Levie, Kimberly; Lewcock, Joseph W.; Di Paolo, Gilbert; Sanchez, Pascal E.

Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice

SCIENCE TRANSLATIONAL MEDICINE

IF14.6

2020-05-27132

PREAI

Ullman, Julie C.; Arguello, Annie; Getz, Jennifer A.; Bhalla, Akhil; Mahon, Cathal S.; Wang, Junhua; Giese, Tina; Bedard, Catherine; Kim, Do Jin; Blumenfeld, Jessica R.; Liang, Nicholas; Ravi, Ritesh; Nugent, Alicia A.; Davis, Sonnet S.; Ha, Connie; Duque, Joseph; Tran, Hai L.; Wells, Robert C.; Lianoglou, Steve; Daryani, Vinay M.; Kwan, Wanda; Solanoy, Hilda; Hoang Nguyen; Earr, Timothy; Dugas, Jason C.; Tuck, Michael D.; Harvey, Jennifer L.; Reyzer, Michelle L.; Caprioli, Richard M.; Hall, Sejal; Poda, Suresh; Sanchez, Pascal E.; Dennis, Mark S.; Gunasekaran, Kannan; Srivastava, Ankita; Sandmann, Thomas; Henne, Kirk R.; Thorne, Robert G.; Di Paolo, Gilbert; Astarita, Giuseppe; Diaz, Dolores; Silverman, Adam P.; Watts, Ryan J.; Sweeney, Zachary K.; Kariolis, Mihalis S.; Henry, Anastasia G.

TREM2 Regulates Microglial Cholesterol Metabolism upon Chronic Phagocytic Challenge

TREM2在慢性吞噬细胞攻击后调节小胶质细胞胆固醇代谢

NEURON

IF15

2020-03-01408

OAAI

Nugent, Alicia A.; Lin, Karin; van Lengerich, Bettina; Lianoglou, Steve; Przybyla, Laralynne; Davis, Sonnet S.; Llapashtica, Ceyda; Wang, Junhua; Kim, Do Jin; Xia, Dan; Lucas, Anthony; Baskaran, Sulochanadevi; Haddick, Patrick C. G.; Lenser, Melina; Earr, Timothy K.; Shi, Ju; Dugas, Jason C.; Andreone, Benjamin J.; Logan, Todd; Solanoy, Hilda O.; Chen, Hang; Srivastava, Ankita; Poda, Suresh B.; Sanchez, Pascal E.; Watts, Ryan J.; Sandmann, Thomas; Astarita, Giuseppe; Lewcock, Joseph W.; Monroe, Kathryn M.; Di Paolo, Gilbert

Novel FACS based method demonstrates CNS cell-type distribution and efficacy of a BBB penetrant ERT in a mouse model of MPS II

MOLECULAR GENETICS AND METABOLISM

IF3.5

2020-02-010

PREAI

SUPT4H1 Depletion Leads to a Global Reduction in RNA

CELL REPORTS

IF6.9

2019-01-0117

OAAI

High-Throughput and Sensitive Quantification of Circulating Tumor DNA by Microfluidic-Based Multiplex PCR and Next-Generation Sequencing

JOURNAL OF MOLECULAR DIAGNOSTICS