Dhruv Sareen

Charting the translational pathway: ISSCR best practices for the development of PSC-derived therapies

Jaqueline Barry, Elsa Abranches, Ricardo P. Baptista, Chris Barry et autres

The successful translation of a stem cell-derived product from the laboratory into an approved medicine requires specific scientific, technical, and regulatory understanding. Since this knowledge base is fragmented across websites and publications, the ISSCR has developed a new resource, The Best Practices …

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TDP-43 loss induces cryptic polyadenylation in ALS/FTD

Sam Bryce-Smith, Anna‐Leigh Brown, Max Z. Y. J. Chien, Dario Dattilo et autres

Nuclear depletion and cytoplasmic aggregation of the RNA-binding protein TDP-43 are cellular hallmarks of amyotrophic lateral sclerosis (ALS). TDP-43 nuclear loss causes de-repression of cryptic exons, yet cryptic alternative polyadenylation (APA) events have been largely overlooked. In this study, we developed a …

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Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes

Rajesh C. Rao, Brigitte L. Arduini, Susan L. Borden, Dhruv Sareen et autres

Retinal pigment epithelium (RPE) cell atrophy in dry age-related macular degeneration (AMD) compromises photoreceptor cell function, leading to vision loss. Stem cell-based RPE replacement therapy aims to reverse disease progression and restore vision. RPESC-RPE-4W, a post-mitotic adult RPE stem cell-derived RPE (RPESC-RPE) …

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Corrigendum to “Comprehensive Proteomics Metadata and Integrative Web Portals Facilitate Sharing and Integration of LINCS Multiomics Data”

D. Vidović, Behrouz Shamsaei, Stephan C. Schürer, Phillip Kogan et autres

Large-scale differentiation of iPSC-derived motor neurons from ALS and control subjects

Michael J. Workman, Ryan G. Lim, Jie Wu, Aaron P. Frank et autres

Publisher of over 50 scientific journals across the life, physical, earth, and health sciences, both independently and in partnership with scientific societies including Cell, Neuron, Immunity, Current Biology, AJHG, and the Trends Journals.

Filamented Light (FLight) Biofabrication of Aligned Fibrillar Structures to Direct 3D Cell Organization Within Microgels

Akshat Joshi, Meenakshi Kamaraj, Nafiseh Moghimi, Hossein Heidari et autres

Controlling cellular organization in hydrogels is of great interest in tissue engineering and regenerative medicine. In the body, cell organization is regulated by aligned extracellular matrices, such as collagen fibers. However, generating patterned extracellular matrix fibers in hydrogels, such as microfabricated gels, …

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Comprehensive Proteomics Metadata and Integrative Web Portals Facilitate Sharing and Integration of LINCS Multiomics Data

D. Vidović, Behrouz Shamsaei, Stephan C. Schürer, Phillip Kogan et autres

The Library of Integrated Network-based Cellular Signatures (LINCS), an NIH Common Fund program, has cataloged and analyzed cellular function and molecular activity profiles in response to >80,000 perturbing agents that are potentially disruptive to cells. Because of the importance of proteins and …

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Identification of Disease-Relevant, Sex-Based Proteomic Differences in iPSC-Derived Vascular Smooth Muscle Cells

Nethika R. Ariyasinghe, Divya Gupta, Sean S. Escopete, Deepika Rai et autres

The prevalence of cardiovascular disease varies with sex, and the impact of intrinsic sex-based differences on vasculature is not well understood. Animal models can provide important insights into some aspects of human biology; however, not all discoveries in animal systems translate well …

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Identification of Disease-relevant, Sex-based Proteomic Differences in iPSC-derived Vascular Smooth Muscle

Nethika R. Ariyasinghe, Divya Gupta, Sean S. Escopete, Aleksandr B. Stotland et autres

The prevalence of cardiovascular disease varies with sex, and the impact of intrinsic sex-based differences on vasculature is not well understood. Animal models can provide important insight into some aspects of human biology, however not all discoveries in animal systems translate well …

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Rare variant analyses validate known ALS genes in a multi-ethnic population and identifies ANTXR2 as a candidate in PLS

Tess D. Pottinger, Joshua E. Motelow, Gundula Povysil, Cristiane Araújo Martins Moreno et autres

Abstract Background Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting over 300,000 people worldwide. It is characterized by the progressive decline of the nervous system that leads to the weakening of muscles which impacts physical function. Approximately, 15% of individuals diagnosed …

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Author Correction: TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A

Anna‐Leigh Brown, Oscar G. Wilkins, Matthew J. Keuss, Sarah E. Hill et autres

The version of the article initially published referred to one, cell line as SK-N-DZ. This line has since been conclusively identified as SK-N-BE(2). The HTML and PDF versions of the article have been updated to reflect this.

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Disease related changes in ATAC-seq of iPSC-derived motor neuron lines from ALS patients and controls

Stanislav Tsitkov, Kelsey Valentine, Velina Kozareva, Aneesh Donde et autres

Amyotrophic Lateral Sclerosis (ALS), like many other neurodegenerative diseases, is highly heritable, but with only a small fraction of cases explained by monogenic disease alleles. To better understand sporadic ALS, we report epigenomic profiles, as measured by ATAC-seq, of motor neuron cultures …

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