Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives

MF Dias, K Joo, JA Kemp, SL Fialho… - Progress in retinal and …, 2018 - Elsevier
Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people
worldwide. It is characterized with progressive loss of rods and cones and causes severe …

Cas9 as a versatile tool for engineering biology

P Mali, KM Esvelt, GM Church - Nature methods, 2013 - nature.com
RNA-guided Cas9 nucleases derived from clustered regularly interspaced short palindromic
repeats (CRISPR)-Cas systems have dramatically transformed our ability to edit the …

A highly photostable and bright green fluorescent protein

M Hirano, R Ando, S Shimozono, M Sugiyama… - Nature …, 2022 - nature.com
The low photostability of fluorescent proteins is a limiting factor in many applications of
fluorescence microscopy. Here we present StayGold, a green fluorescent protein (GFP) …

A third-generation lentivirus vector with a conditional packaging system

T Dull, R Zufferey, M Kelly, RJ Mandel… - Journal of …, 1998 - Am Soc Microbiol
Vectors derived from human immunodeficiency virus (HIV) are highly efficient vehicles for in
vivo gene delivery. However, their biosafety is of major concern. Here we exploit the …

Visualizing spatiotemporal dynamics of multicellular cell-cycle progression

A Sakaue-Sawano, H Kurokawa, T Morimura, A Hanyu… - Cell, 2008 - cell.com
The cell-cycle transition from G 1 to S phase has been difficult to visualize. We have
harnessed antiphase oscillating proteins that mark cell-cycle transitions in order to develop …

[HTML][HTML] Gene therapy-promises, problems and prospects

IM Verma, N Somia - Nature, 1997 - nature.com
The concept behind gene therapy is simple—by delivering corrective genetic material to the
cells of a patient the symptoms of disease can be alleviated. But seven years after the first …

Development of a self-inactivating lentivirus vector

H Miyoshi, U Blömer, M Takahashi, FH Gage… - Journal of …, 1998 - Am Soc Microbiol
We have constructed a new series of lentivirus vectors based on human immunodeficiency
virus type 1 (HIV-1) that can transduce nondividing cells. The U3 region of the 5′ long …

Electroporation and RNA interference in the rodent retina in vivo and in vitro

T Matsuda, CL Cepko - Proceedings of the National …, 2004 - National Acad Sciences
The large number of candidate genes made available by comprehensive genome analysis
requires that relatively rapid techniques for the study of function be developed. Here, we …

Histone–GFP fusion protein enables sensitive analysis of chromosome dynamics in living mammalian cells

T Kanda, KF Sullivan, GM Wahl - Current Biology, 1998 - cell.com
Background: The amplification of oncogenes in cancer cells is often mediated by paired
acentric chromatin bodies called double minute chromosomes (DMs), which can accumulate …

Wnt-mediated activation of NeuroD1 and retro-elements during adult neurogenesis

T Kuwabara, J Hsieh, A Muotri, G Yeo… - Nature …, 2009 - nature.com
In adult hippocampus, new neurons are continuously generated from neural stem cells
(NSCs), but the molecular mechanisms regulating adult neurogenesis remain elusive. We …