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#基因治疗/编辑

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Oct 15Thu
  1. biospace.com62

    FDA's latest rare disease framework seeks to prioritize consistency in flexibility

    The FDA launched its Rare Disease Framework Initiative to standardize how clinical trial requirements are applied across rare diseases while tailoring evidence expectations to what small patient populations can realistically generate. Only 5% of an estimated 10,000 rare conditions affecting 30 million Americans have an FDA-approved treatment. The framework builds on programs such as the Rare Disease Innovation Hub, CDER's ARC program and the RDEA pilot, and follows the November 2025 plausible mechanism pathway. The FDA will hold a meeting on Oct. 28 to gather feedback from patients, advocates and industry.

    Why it matters: FDA policy shift sets consistent rules for rare disease drug development, a core issue for sponsors of gene and cell therapies.

Oct 14Wed
  1. Mol Cell64

    Structural basis of directionality control in large serine integrases.

    Eight structures of SPβ integrase-DNA complexes, at resolutions down to 3.15 Å, reveal how directionality is controlled along the integrative and excisive pathways. Recombination-induced conformational changes and RDF-mediated repositioning of the coiled-coil subdomain determine which DNA site pairs form active synaptic complexes and which product complexes become conformationally locked against back reactions. The excision synaptic complex is fundamentally different from the integration one, providing a framework for engineering improved genome editing tools.

  2. biorxiv.org50

    Biomarker-driven and gene-enhanced viral therapy for malignant peripheral nerve sheath tumor

    Loss of the PRC2 histone mark H3K27me3, common in high-grade MPNST, sensitizes tumors to the oncolytic HSV G47Δ, but also triggers a compensatory integrated stress response that blunts viral protein translation. Engineers built G47Δ² with tumor-regulated ICP34.5 expression (BIRC5-driven), limiting daily xenograft tumor-volume growth to 0.6% (95% CI -2.9–4.2%) versus 8.3% for G47Δ² and 22.5% for controls, though murine gastrointestinal toxicity reduced event-free survival.

Oct 13Tue
  1. Nat Biotechnol72

    Discovery and engineering of retrons for precise genome editing.

    Engineered retron reverse transcriptases identified from metagenomic data produce single-stranded DNA donors in mammalian cells at efficiencies comparable to conventional oligodeoxynucleotide donors, but from a genetically encoded cassette. The editors work with Cas12a nuclease and Cas9 nickase, broadening targetable sites and avoiding double-stranded breaks. An all-RNA delivery strategy enables DNA-free editing in cells and vertebrate embryos, demonstrated by inserting a split GFP epitope tag for live-cell imaging.

    Why it matters: It turns retrons into genetically encoded donor systems for precise editing without exogenous DNA or double-strand breaks.

  2. nature.com60

    Tracing the origins of CRISPR–Cas

    VIPR systems, a family of RNA-guided DNA-targeting systems found mainly in bacteriophages, may be evolutionary precursors to class 1 CRISPR-Cas. Yoon and colleagues identified them through structure-based searches of the AlphaFold database and showed that small Vipr proteins bind VIPR RNAs with tandem repeats. The NN bases guide DNA targeting through a 'one-nucleotide skip' code, validated in vitro and by transcriptional repression in Escherichia coli, and reprogramming the NN sequences redirects targeting without a protospacer-adjacent motif.

    Why it matters: It points to evolutionary precursors of CRISPR-Cas and a targeting rule that could enable new programmable genetic manipulation tools.

Oct 12Mon
  1. Sci Adv64

    SMCHD1 is a target for gene activation therapy to treat Prader-Willi syndrome.

    SMCHD1 represses the entire Prader-Willi syndrome locus in neural lineages in both mice and humans, making it a candidate target for activating the silenced maternal copy of the disease genes. Deleting Smchd1 after early development in vivo activated PWS genes in disease-relevant mouse tissues including hypothalamus and improved phenotypes in a PWS mouse model. Genome-wide SMCHD1 binding aside, this postearly targeting appeared safe, supporting gene activation therapy for the underlying cause of PWS rather than individual symptoms.

    Why it matters: It identifies a molecular target that activates the silenced maternal allele and improves disease phenotypes in a PWS mouse model.

Oct 11Sun
  1. biorxiv.org70

    A unified framework for quantitative splicing and transcript prediction

    SpliceAI2 is a deep learning model trained end-to-end on splice site, splice junction, and transcript usage data across multiple mammalian species, and it reconstructs complete transcripts directly from genomic sequence. It improves detection and quantification of splice-altering variants across benchmarks covering cryptic splicing, splicing QTLs, and massively parallel reporter assays. In a rare disease cohort, predicted splice-altering variants account for 15% of the excess genetic burden, with about half in deep intronic regions likely missed by exome sequencing.

    Why it matters: SpliceAI2 quantifies splice-altering variant effects genome-wide, including deep intronic variants exome sequencing misses.

Oct 9Fri
  1. Sci Adv63

    Toward transgenic multicellular parasites: CRISPR-Cas12a germline editing of Schistosoma mansoni eggs as key to success.

    Cas12a outperformed Cas9 for homology-directed insertion of a 5'-C6-PEG10-modified donor template with 50-nucleotide microhomology arms into the GSH1 safe-harbor site of Schistosoma mansoni eggs. Electroporated eggs expressing EGFP under an S. mansoni ubiquitin promoter yielded transgenic cercariae that produced EGFP-positive adult worms in hamsters. Sequencing confirmed stable chromosomal integration transmitted through filial generations, opening functional genomics in platyhelminths.

  2. biospace.com59

    Reusing data could cut R&D costs and greatly increase gene editing’s reach: ARM

    The Alliance for Regenerative Medicine (ARM) has published a playbook, building on FDA draft guidance from this summer, showing how gene-editing developers can reuse toxicity, manufacturing and other data across a platform to cut redundant studies and costs. Beam Therapeutics, which helped create the playbook, designs Phase 1 trials as registrational and aims to move from first-in-human studies to FDA approval in four to five years, versus eight to 10 traditionally. ARM also calls for a bifurcated regulatory framework allowing higher-risk, streamlined development for severe rare diseases with no standard of care, while William Blair analysts say more clarity on the plausible mechanism pathway is needed.

  3. doi.org40

    Innovative niosome screening for efficient gene delivery in senescent primary umbilical cord stroma-derived MSCs

    DP20CQ niosomes, built with DOTMA and polysorbate 20 plus chloroquine, delivered genes more effectively than DP80CH formulations in Palbociclib-induced senescent UC-MSCs from four donors. At DOTMA/DNA ratios of 10/1 and 15/1, DP20CQ achieved the highest transgene expression in senescent UC-MSCs, surpassing Lipofectamine, with strong endosomal escape at 10/1 and no loss of cell viability. This positions niosomes as gene delivery vehicles for senescent cells in antiaging therapy.

  4. biopharmadive.com52

    In the hunt for reverse merger targets, Caribou could be prized game

    Caribou Biosciences, the CRISPR company co-founded by Jennifer Doudna, will stop developing its two off-the-shelf cell therapy programs for blood malignancies, citing a financing environment that CEO Rachel Haurwitz called increasingly challenging. The board has cleared management to evaluate a merger, acquisition or other transactions, with Wedbush Securities as exclusive financial advisor. Caribou ended the second quarter with $114 million in cash and securities, and Leerink analyst Daina Graybosch sees a reverse merger or a sale to Tang Capital as likely outcomes.

    Why it matters: A prominent CRISPR biotech with $114 million in cash is winding down and auctioning itself amid a wave of biotech reverse mergers.

Oct 8Thu
  1. fda.gov41

    Recently Issued Guidance Documents

    The FDA has released a set of guidance documents for industry and agency staff issued from July to September 2026. The list includes final guidances on blood collection and storage systems for blood components using the buffy coat method, formal PDUFA meetings, cancer clinical trial eligibility criteria on laboratory values, washout periods and performance status, and FAQs for cellular and gene therapy products. Draft guidances cover premarket approval electronic submission templates, potency assessment of active immunotherapy products, container closure systems for drugs, biologicals and biosimilars.

  2. Nat Biotechnol69

    RNA structure modulates Cas13 activity and enables mismatch detection.

    Secondary structure within the protospacer and immediately 3' to it inhibits Cas13 activity, an effect explained quantitatively by a strand displacement framework. Harnessing strand displacement to create an 'occluded' Cas13 enhances mismatch discrimination up to 50-fold and detects sequence-agnostic mutations at allele frequencies below 1%. The approach identified human-adaptive mutations in SARS-CoV-2 and influenza A viruses and oncogenic KRAS mutations.

    Why it matters: It turns Cas13's structural sensitivity into a 50-fold mismatch discrimination gain for low-frequency mutation detection.

Oct 7Wed
  1. biospace.com40

    Genmab and AbbVie’s Epkinly win, a rush of deals, Sarepta’s turnaround hopes, more

    Genmab and AbbVie's bispecific antibody Epkinly, approved in May 2023 for diffuse large B cell lymphoma after at least two prior systemic lines, cut risk of disease progression or death by 51% in a Phase 3 trial when combined with R-CHOP in the frontline setting. In deals, Shionogi bought IntraBio for $2 billion, Novartis committed up to $7.8 billion to Abogen Biosciences, Eli Lilly added up to $870 million with Gate Bioscience, and Sanofi and Regeneron renewed their collaboration with a $1 billion upfront. Sarepta shared Elevidys data in older Duchenne patients at the World Muscle Society meeting, with Jefferies citing upcoming readouts and an FDA decision on Amondys 45 and Vyondys 53.

Oct 6Tue
  1. Nat Biotechnol66

    Discovery and engineering of avian R2 retrotransposons for all-RNA-mediated targeted DNA integration in human cells.

    Screening 1,139 avian genomes identified 159 avian R2 retrotransposons, whose sequence features were characterized. Engineered variants of these retrotransposons achieved up to 60% site-specific gene integration across human primary cells. The work expands the avian R2 resource and adds tools for all-RNA-mediated genome editing.

    Why it matters: High-efficiency targeted DNA integration with all-RNA delivery could broaden genome-editing options.

  2. biocentury.com80

    Nobel Prize for optogenetics arrives as first therapy nears FDA decision

    Karl Deisseroth, Peter Hegemann and Georg Nagel share the 2026 Nobel Prize in Physiology or Medicine for discoveries concerning light-gated ion channels and optogenetics. FDA is due to decide by mid-2027 whether the first optogenetic therapy, a retinal gene therapy, reaches the market. Nanoscope leads direct therapeutic applications of the technology, while MapLight uses it to guide psychiatric drug discovery.

    Why it matters: Nobel recognition coincides with the first optogenetic therapy approaching an FDA decision.

Oct 5Mon
  1. nature.com73

    Gene therapy for rare lysosomal storage disorder nabs FDA approval

    The FDA has approved Ultragenyx Pharmaceutical's gene therapy rebisufligene etisparvovec (Fayuvi) for paediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. It is the first FDA-approved treatment option for this rare neurodegenerative lysosomal storage disorder.

    Why it matters: First FDA-approved therapy for a rare neurodegenerative lysosomal storage disorder.

  2. nature.com70

    Discovery and engineering of avian R2 retrotransposons for all-RNA-mediated targeted DNA integration in human cells

    Avian R2 retrotransposons were discovered and engineered as an all-RNA-mediated system for targeted DNA integration in human cells. Sequencing data from the work were deposited in the Genome Sequence Archive for Human under accession HRA013312, and the analytic code is publicly available on GitHub. The approach offers an RNA-only route to site-specific DNA insertion, in contrast to existing DNA-cleavage-based integration methods.

    Why it matters: A new avian R2 system extends all-RNA targeted DNA integration to human cells.

Oct 2Fri
  1. Nature67

    Structural mechanism governing the directionality of bridge recombination.

    IS621 recombinase-bridge RNA complexes mediate excision less efficiently than insertion, and cryo-EM structures explain why: excision substrates adopt linear conformations forming an X-shaped structure, unlike the bent U-shape of insertion, which reduces top-strand exchange efficiency and explains the natural bias toward insertion. Both reactions are tuned by base pairing between bRNA handshake-guide dinucleotides and top strands. The work clarifies the full IS110 transposition cycle and guides design of programmable bridge-editing tools.

  2. nature.com61

    Reactivation of the embryonic ζ-globin gene ameliorates severe forms of α-thalassemia

    Reactivating the embryonic ζ-globin gene can ameliorate severe forms of α-thalassemia. Liu et al. identify cis-regulatory elements that silence ζ-globin in definitive erythroid cells, and gene-editing strategies targeting them restore ζ-globin expression to therapeutically relevant levels in mouse and human disease models. The work points to a new genome-editing approach for severe α-thalassemia.

    Why it matters: It identifies cis-regulatory silencers of ζ-globin and shows gene editing can reactivate it in disease models.

Oct 1Thu
  1. BioCentury43

    Beam lawsuit accuses YolTech and Serapha of tech theft

    Beam Therapeutics has sued YolTech and Serapha Bio, alleging trade secret theft. The suit, filed Sept. 25, claims former Beam scientist Zi Jun (Emma) Wang secretly co-founded YolTech while at Beam and used Beam technology in YolTech's lipid patent application and alpha-1 antitrypsin deficiency program, later licensed to Serapha, where Wang serves as CTO. YolTech CEO Yuxuan Wu categorically rejects the accusations.

  2. Nat Biotechnol50

    Cargo size matters when designing lipid nanoparticles for delivery of large gene editors.

    Screening ionizable lipids with a gene-editor-sized RNA yielded LC-1, a lipid that forms a structure enhancing endosomal escape of large editor mRNAs. Lipid nanoparticles built on LC-1 achieved efficient genome editing in the liver, lung and brain of mice. The work points to cargo size as a design criterion for lipid nanoparticles delivering large gene editors.

    Why it matters: It identifies an ionizable lipid, LC-1, that extends LNP genome editing to liver, lung and brain in mice.

  3. nature.com56

    Restoring hearing in people with a rare form of deafness

    A gene therapy delivering a functional OTOF gene to inner-ear hair cells improved hearing in around 80% of participants within 24 weeks in the CHORD clinical trial, which enrolled 24 participants aged between 10 months and 16 years with OTOF-associated hearing impairment. Each participant received Otarmeni as a one-time infusion, either in one ear or in both ears. In April, Otarmeni became the first gene therapy for hearing loss approved by the US Food and Drug Administration.

Sep 30Wed
  1. nature.com70

    Intravenous hyaluronidase-expressing oncolytic adenovirus with chemotherapy in metastatic pancreatic cancer: a randomized phase 2b trial

    Intravenous zabilugene almadenorepvec (VCN-01), a hyaluronidase-expressing oncolytic adenovirus, plus gemcitabine and nab-paclitaxel prolonged overall survival over chemotherapy alone in the randomized phase 2b VIRAGE trial in treatment-naive metastatic pancreatic cancer. The finding supports adding an oncolytic virus to standard first-line chemotherapy in this setting.

    Why it matters: First randomized evidence that an intravenous oncolytic adenovirus improves survival in metastatic pancreatic cancer.

  2. biospace.com45

    Milestone Sanfilippo nod helps Ultragenyx regroup after tough Angelman defeat

    The FDA approved Ultragenyx's Fayuvi for Sanfilippo syndrome type A, the first-ever treatment for that condition, shortly after approving the company's first gene therapy, Genglycos, for glycogen storage disease type Ia; each approval came with a priority review voucher worth as much as $200 million. The wins follow the Phase 3 Asire trial failure of apazunersen in Angelman syndrome, which cut the stock nearly 50%, and a January failure of setrusumab in osteogenesis imperfecta that erased $1 billion in value. CEO Emil Kakkis says Ultragenyx will implement significant expense reductions while keeping the setrusumab program and awaiting an upcoming Angelman readout.

    Why it matters: Two rare-disease approvals including a first Sanfilippo treatment come amid Phase 3 failures forcing cost cuts at Ultragenyx.

Sep 28Mon
  1. nature.com59

    Germline homologous recombination deficiency influences TP53 -mutant clonal hematopoiesis fitness during platinum and PARP inhibitor treatment

    Platinum chemotherapy drives DNA damage response gene-mutant clonal hematopoiesis more strongly than PARP inhibitors, an analysis of treatment-associated clone behavior shows. Carrying germline mutations that confer homologous recombination deficiency slowed the expansion of these clones during treatment. The findings separate the mutagenic pressure of the two drug classes and identify germline HR status as a modifier of TP53-mutant clone fitness.

    Why it matters: It distinguishes platinum from PARP inhibitor effects on clonal hematopoiesis and adds germline HR status as a modifier of clone expansion.

  2. nature.com45

    Cargo size matters when designing lipid nanoparticles for delivery of large gene editors

    Ionizable lipids screened with a gene-editor-sized messenger RNA yielded LC-1, a lipid that forms nanoparticles with a structure enhancing endosomal escape. LC-1 enabled efficient genome editing in the liver, lung and brain of mice, addressing the reduced performance lipid nanoparticles often show when carrying the large messenger RNAs that encode gene editors.

Sep 27Sun
  1. biorxiv.org54

    Targeted CRISPRi screening reveals unexpected resilience across the RNA polymerase III transcriptome

    An inducible CRISPR interference platform with a custom sgRNA library enabled gene-specific repression of RNA polymerase III-transcribed genes and Pol III machinery. Genome-wide screens in diploid fibroblasts and HEK293T cells uncovered several Pol III dependencies, with multiple initiator methionine tRNA genes among the strongest fitness dependencies. Glioblastoma models, however, remained largely insensitive to repression of both individual Pol III genes and core Pol III components despite efficient target knockdown, challenging the assumption that cancer cells uniformly depend on elevated Pol III activity.

    Why it matters: It shows glioblastoma models tolerate Pol III gene repression, contradicting the link between Pol III activity and cancer growth.

Sep 24Thu
  1. biorxiv.org42

    Convergent evolutionary loss of chemosensory and blood-feeding pathways in non-blood-feeding mosquitoes

    Comparing chromosome-level genomes and whole-brain reconstructions across seven mosquito species, including three independent non-blood-feeding lineages, reveals convergent loss of blood-feeding genes: Aegyptin, blood-activated serine proteases Chymotrypsin-1 and 2, a carboxylesterase, and multiple chemosensory receptor clades. Non-blood-feeding females also show reduced female-biased head gene expression and smaller antennal lobes. The work defines a compact gene set underlying a complex trait lost three times over roughly 200 million years.

    Why it matters: Convergent losses across three lineages pinpoint the genes and brain regions required for mosquito blood feeding.

Sep 19Sat
  1. BioCentury69

    Ultragenyx wins first approval for MPS IIIA, closing a bumpy four-year path

    FDA granted Ultragenyx traditional approval for rebisufligene etisparvovec, now Fayuvi, the first treatment for Sanfilippo syndrome Type A (MPS IIIA). CBER approved the gene therapy on clinical evidence that treated children maintained or improved cognitive function versus an untreated natural-history cohort, rather than on CSF heparan sulfate as a surrogate endpoint. The outcome exceeds the accelerated approval Ultragenyx had sought in its original application and resubmission, while CBER's biomarker stance remains unresolved.

    Why it matters: First therapy approved for MPS IIIA and an FDA traditional approval relying on external controls.

Sep 18Fri
  2. fda.gov70

    FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A

    The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time intravenous AAV9 gene therapy from Ultragenyx Pharmaceutical, as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A. In an open-label, single-arm multicenter study, treated patients aged 2 to 5 maintained or improved cognitive scores versus an untreated historical control. Patients receive corticosteroids from one day before infusion through at least eight weeks after, with warnings including thrombotic microangiopathy.

    Why it matters: First FDA-approved therapy for a rare pediatric neurodegenerative disease with no prior disease-altering treatment.

Sep 15Tue
  1. Sci Adv64

    Engineering suppressor tRNAs for effective treatment of Duchenne muscular dystrophy.

    Engineered UAA suppressor tRNAs, delivered systemically with optimized AAV vectors and RNA polymerase III promoters, restored full-length dystrophin and recovered muscle strength and motor function in mice with nonsense mutations, at lower doses than AAV microdystrophin therapies. No adverse toxicology appeared, and proteome-wide analysis showed reversion of molecular pathophysiology. The approach targets the roughly 15% of DMD patients with nonsense mutations.

    Why it matters: First platform shown to restore full-length dystrophin rather than a truncated version, at lower doses than microdystrophin.

Sep 14Mon
  1. Nature69

    Creating bottom-up RNA transfer vehicles from synthetic protein assemblies.

    AI-designed protein assemblies combined with natural protein domains yielded more than 100 bottom-up RNA transfer vehicles with unique sizes and shapes, surpassing the RNA transfer efficiency of widely used delivery vehicles by several orders of magnitude. Tropism was programmed with computationally designed peptide binders, enabling delivery of therapeutically relevant cargo RNAs across cellular models. One vehicle's in vivo biodistribution was mapped in a mouse at near-single-cell resolution, confirmed as safe, and used for a gene-editing treatment strategy for Duchenne muscular dystrophy in patient-derived cells and a pig.

    Why it matters: Generative AI-designed protein assemblies outperform established RNA delivery vehicles and work in vivo.

  2. Sci Adv48

    Widespread atypical UV-induced mutations form in single-stranded DNA.

    UV light induces an extended array of noncanonical mutations in single-stranded DNA, including AT>AM, GT>GV, AC>AA, AT>TT, and TA>TT substitutions that resist photo-reversion and likely originate from atypical photoproducts. The work coupled temperature-induced telomeric end resection in yeast with serial UV irradiation and whole-genome sequencing. These ssDNA-specific lesions offer an additional damage-mediated route to oncogenic melanoma mutations such as BRAF V600E, which involves a GT>GA substitution.

    Why it matters: It explains how UV light can generate melanoma-driver mutations that canonical SBS7-type lesions do not account for.

Sep 11Fri
  1. Nat Biotechnol53

    Programmable promoter editing for precise control of transgene expression.

    DIAL is a modular framework for building editable promoters that tune transgene expression in fine, heritable steps. Recombinase-mediated excision of nested spacers between zinc finger transcription factor binding sites and the core promoter generates a tunable range of unimodal expression setpoints from a single promoter, with small-molecule temporal control. Lentiviral delivery produced multiple setpoints in primary cells and induced pluripotent stem cells, and enabled mapping of transgene levels to phenotype in direct conversion to induced motor neurons.

    Why it matters: Gives a general method for titrating and locking in transgene expression levels, useful wherever gene dosage matters.

  2. Nat Biotechnol56

    Engineered genomic attachment sites for site-specific recombinases enable high-efficiency integration in plants and human cells.

    Rationally engineered chimeric attachment sites boost Bxb1-mediated integration efficiency in human cells and plants. The attB(V111) variant achieved 51.9% integration in HEK293T cells (1.7-fold over wild-type attB) and 35.6% in rice protoplasts (4.4-fold), and paired with a Bxb1 catalytic-domain mutant delivered 31% integration of a CD19 CAR cassette and 25% of an ornithine transcarbamylase cassette in human cells. In rice, a 5.8 kb herbicide-resistance cassette integrated in 24% of regenerated plants, with heritable transmission to T1 plants.

    Why it matters: Engineered attachment sites substantially raise recombinase-mediated integration efficiency across plants and human cells.

Sep 10Thu
  1. Nature67

    Efficient and precise programmable DNA knock-in without double-strand breaks.

    CRISPR kilobase-scale nickase-targeting (KNIT) editing inserts DNA fragments from 0.7 kb to over 10 kb without double-strand DNA cleavage by coupling a Cas9 nickase with a DNA donor recruiting system. It achieves up to 89% efficiency, cuts indel rates, translocations and off-target editing, restores gene expression in mutant cells, and enables non-viral CAR-T engineering with antitumour activity in vitro and in mice. This shifts kilobase-scale knock-in away from double-strand-break editing.

    Why it matters: A break-free CRISPR method reaching 89% knock-in efficiency could change how cell therapies are engineered.

Sep 9Wed
  1. Nat Biotechnol67

    Design of optimized epigenetic regulators for durable gene silencing with application to PCSK9 in nonhuman primates.

    An optimized TALE-based epigenetic regulator, EpiReg-T, silenced PCSK9 in macaque liver with over 90% efficiency lasting 343 days after a single lipid nanoparticle dose, lowering LDL cholesterol. In mice, EpiReg-T reached 98% efficiency versus 64% for the initial dCas9-based design. Multiomic analyses showed minimal off-target effects in monkeys, mice and human-derived cells, and the platform can be retargeted to other genes by reengineering the DNA-binding domain.

    Why it matters: Durable, single-dose epigenetic PCSK9 silencing with minimal off-target effects in nonhuman primates moves epigenetic editing toward the clinic.

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