Suchergebnisse - "Lek, Angela"

PubPharm (38)

1

COMPOSITIONS AND METHODS FOR UPREGULATING ISOFORMS OF DYSTROPHIN AS THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY (DMD)

enthalten in: Europäisches Patentamt | 2024

von LEK MONKOL | LEK ANGELA | WOODMAN KERYN | +1

2

Profilin1 is required for prevention of mitotic catastrophe in murine and human glomerular diseases

enthalten in: The Journal of clinical investigation | 2023

von Tian, X. | Pedigo, C. | Li, K. | +18

3

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. Reply

enthalten in: The New England journal of medicine | 2023

von Lek, A. | Keeler, A. | Flotte, T.

CommentOn: N Engl J Med. 2023 Sep 28;389(13):1203-1210. - PMID 37754285

4

Deep Mutational Scanning in Disease-related Genes with Saturation Mutagenesis-Reinforced Functional Assays (SMuRF)

enthalten in: bioRxiv.org | 2023

von Ma, K. | Ng, K. | Huang, S. | +12

5

Deep Mutational Scanning in Disease-related Genes with Saturation Mutagenesis-Reinforced Functional Assays (SMuRF)

enthalten in: bioRxiv : the preprint server for biology | 2023

von Ma, K. | Ng, K. | Huang, S. | +12

6

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy

enthalten in: The New England journal of medicine | 2023

von Lek, A. | Wong, B. | Keeler, A. | +22

CommentIn: N Engl J Med. 2023 Dec 7;389(23):2210-2211. - PMID 38055267

7

Compositions and methods for treating facioscapulohumeral dystrophy

enthalten in: Europäisches Patentamt | 2023

von LEK ANGELA | KUNKEL LOUIS M

8

Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator

enthalten in: bioRxiv.org | 2023

von Lek, A. | Wong, B. | Keeler, A. | +22

9

Flavones provide resistance to DUX4-induced toxicity via an mTor-independent mechanism

enthalten in: Research square | 2023

von Cohen, J. | Huang, S. | Koczwara, K. | +6

UpdateIn: Cell Death Dis. 2023 Nov 16;14(11):749. - PMID 37973788

10

Development of Assays to Measure GNE Gene Potency and Gene Replacement in Skeletal Muscle

enthalten in: Journal of neuromuscular diseases | 2023

von Zygmunt, D. | Lam, P. | Ashbrook, A. | +4