Gene-editing in patient and humanized-mice primary muscle stem cells rescues dysferlin expression in dysferlin-deficient muscular dystrophy.

Dystrophy-associated fer-1-like protein (dysferlin) conducts plasma membrane repair. Mutations in the DYSF gene cause a panoply of genetic muscular dystrophies. We targeted a frequent loss-of-function, DYSF exon 44, founder frameshift mutation with mRNA-mediated delivery of SpCas9 in combination with a mutation-specific sgRNA to primary muscle stem cells from two homozygous patients.

Disintegration of the NuRD Complex in Primary Human Muscle Stem Cells in Critical Illness Myopathy.

Critical illness myopathy (CIM) is an acquired, devastating, multifactorial muscle-wasting disease with incomplete recovery. The impact on hospital costs and permanent loss of quality of life is enormous. Incomplete recovery might imply that the function of muscle stem cells (MuSC) is impaired.

Co-Regulated Gene Expression as a Suitable Readout after Precise Gene Correction.

LMNA-related muscular dystrophy is an autosomal-dominant progressive disorder caused by mutations in LMNA. LMNA missense mutations are becoming correctable with CRISPR/Cas9-derived tools. Evaluating the functional recovery of LMNA after gene editing bears challenges as there is no reported direct loss of function of lamin A/C proteins in patient-derived cells.

Generation of hiPSC-Derived Skeletal Muscle Cells: Exploiting the Potential of Skeletal Muscle-Derived hiPSCs.

Cell therapies for muscle wasting disorders are on the verge of becoming a realistic clinical perspective. Muscle precursor cells derived from human induced pluripotent stem cells (hiPSCs) represent the key to unrestricted cell numbers indispensable for the treatment of generalized muscle wasting such as cachexia or intensive care unit (ICU)-acquired weakness. We asked how the cell of origin influences efficacy and molecular properties of hiPSC-derived muscle progenitor cells.

The Lepidoptera of Cuatrociénegas Protected Area 1. A new species in the genus Hübner, [1823] (Erebidae, Erebinae, Euclidiini) from the Chihuahuan Desert, Coahuila, Mexico.

A new species of Hübner, [1823] (Lepidoptera, Erebidae, Erebinae, Euclidiini) is described from Cuatrociénegas Protected Area and Biosphere Preserve in Coahuila, Mexico. Adult male and female moths are illustrated, including genitalia. Homziak & Metzler, is one of 27 new species of insects discovered during an inventory survey of arthropods of White Sands National Monument, USA, and Cuatrociénegas Protected Area (Mexico), funded by the U.

Base editing repairs an SGCA mutation in human primary muscle stem cells.

Skeletal muscle can regenerate from muscle stem cells and their myogenic precursor cell progeny, myoblasts. However, precise gene editing in human muscle stem cells for autologous cell replacement therapies of untreatable genetic muscle diseases has not yet been reported. Loss-of-function mutations in SGCA, encoding α-sarcoglycan, cause limb-girdle muscular dystrophy 2D/R3, an early-onset, severe, and rapidly progressive form of muscular dystrophy affecting both male and female patients.

Generation of two human induced pluripotent stem cell lines derived from myoblasts (MDCi014-A) and from peripheral blood mononuclear cells (MDCi014-B) from the same donor.

We describe the generation and characterization of two human induced pluripotent stem cell (hiPSCs) lines reprogrammed from myoblasts and from peripheral blood mononuclear cells (PBMCs) from the same donor. The donor was free of neuromuscular disorders, male and 18 years of age. For reprogramming we used Sendai-virus delivery of the four Yamanaka factors.

Generation of three age and gender matched pairs of human induced pluripotent stem cells derived from myoblasts (MDCi011-A, MDCi012-A, MDCi013-A) and from peripheral blood mononuclear cells (MDCi011-B, MDCi012-B, MDCi013-B) from the same donor.

We describe the generation and characterization of three pairs of human induced pluripotent stem cell (hiPSC) lines reprogrammed from myoblasts and from peripheral blood mononuclear cells (PBMCs) of the same donor. All donors were free of neuromuscular disorders, female and between 47 and 50 years of age. For reprogramming we used Sendai-virus delivery of the four Yamanaka factors.

The Lepidoptera of White Sands National Monument, Otero County, New Mexico, USA 9. A new species of Walker (Cossidae, Hypoptinae) dedicated to Delinda Mix, including a list of species of Cossidae recorded from the Monument.

The U.S. National Park Service initiated a 10-year study of the Lepidoptera at White Sands National Monument, Otero County, New Mexico in late 2006.

The Lepidoptera of White Sands National Monument, Otero County, New Mexico, USA 10. A remarkable new white species of Chionodes Hübner (Gelechiidae).

The U.S. National Park Service initiated a 10-year study, in late 2006, of the Lepidoptera at White Sands National Monument, Otero County, New Mexico.

A review of the genus Ogdoconta Butler (Lepidoptera, Noctuidae, Condicinae, Condicini) from North America north of Mexico with descriptions of three new species.

The species of the genus Ogdoconta Butler, 1891 (Lepidoptera, Noctuidae, Condicinae, Condicini) from North America north of Mexico are reviewed, and a description of the genus is given. Ogdoconta satana Metzler, Knudson & Poole, sp. n.

The Lepidoptera of White Sands National Monument, Otero County, New Mexico, USA 4. A new species of Schinia Hübner, 1818 (Lepidoptera, Noctuidae, Heliothinae).

In 2006 the U.S. National Park Service initiated a long term study of the Lepidoptera at White Sands National Monument, Otero County, New Mexico.

The Lepidoptera of White Sands National Monument, Otero County, New Mexico, USA 3. A new species of Aleptina Dyar, 1902 (Lepidoptera, Noctuidae, Amphipyrinae, Psaphidini).

In 2006 the US National Park Service initiated a long-term study of the Lepidoptera at White Sands National Monument, Otero County, New Mexico. Aleptina arenariasp. n.

The Lepidoptera of White Sands National Monument, Otero County, New Mexico, USA 2. Rediscovery and description of Sparkia immacula (Grote, 1883) (Noctuidae, Noctuinae, Hadenini).

In 2006 the U.S. National Park Service initiated a long term study of the Lepidoptera at White Sands National Monument, Otero County, New Mexico.

A new species of Elasmia Möschler from New Mexico and Texas, and a new subspecies of Elasmia mandela (Druce) from Texas and Oklahoma (Lepidoptera, Notodontidae, Nystaleinae).

Hippia packardii (Morrison) and Hippia insularis (Grote) are moved to the genus Elasmia Möschler as comb. n.Elasmia cave Metzler,sp.